2020
澳门正规博彩十大排行平台 Expands Vosoritide Clinical Program
澳门正规博彩十大排行平台 announced in November that the Company is expanding its clinical program for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), with two new Phase 2 studies. The first study is sponsored by 澳门正规博彩十大排行平台 to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression. The second study is an investigator-initiated study sponsored by Children’s National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.
Food and Drug Administration Accepts 澳门正规博彩十大排行平台’s New Drug Application for Vosoritide to Treat Children with Achondroplasia
澳门正规博彩十大排行平台 announced in November that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. This acceptance by the 澳门十大博彩正规游戏平台 marks the first marketing application accepted for a treatment for achondroplasia in the United States.
澳门正规博彩十大排行平台 Receives 澳门十大博彩正规游戏平台 Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection for Treatment of Adults with PKU
澳门正规博彩十大排行平台 announced in October that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) has approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of 60 mg with Palynziq® (pegvaliase-pqpz) Injection for treatment of adults with Phenylketonuria (PKU). Previously, the maximum dose was 40 mg. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.
Scientific Luminary and MacArthur Fellowship Winner, Kevin Eggan, Ph.D., Joins 澳门正规博彩十大排行平台 as Head of Research and Early Development
澳门正规博彩十大排行平台 announced in October the Company hired scientific luminary and MacArthur Fellowship winner, Kevin Eggan, Ph.D., as Group Vice President, Head of Research and Early Development, effective today. Dr. Eggan brings almost two decades of experience in groundbreaking scientific research in rare neurological disorders.
澳门正规博彩十大排行平台 Receives 澳门十大博彩正规游戏平台 Fast Track Designation for PKU Investigational Gene Therapy, BMN 307
澳门正规博彩十大排行平台, a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced in October that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) has granted Fast Track designation to BMN 307, an investigational gene therapy for the treatment of individuals with PKU.
澳门正规博彩十大排行平台 Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy
澳门正规博彩十大排行平台 announced in September that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.
澳门正规博彩十大排行平台 Announces Presentation of Vosoritide Phase 3 Data in Children with Achondroplasia at the American Society for Bone and Mineral Research 2020 Annual Meeting
澳门正规博彩十大排行平台 announced in September that Lynda Polgreen, MD, MS, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA will present data from the randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET. Achondroplasia is the most common form of disproportionate short stature in humans.
澳门正规博彩十大排行平台 Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia
澳门正规博彩十大排行平台 announced in September that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide. The data demonstrated that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles.
澳门正规博彩十大排行平台 Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia
澳门正规博彩十大排行平台 announced in August that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for vosoritide.
澳门正规博彩十大排行平台 Receives Complete Response Letter (CRL) from 澳门十大博彩正规游戏平台 for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
澳门正规博彩十大排行平台 announced in August that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The 澳门十大博彩正规游戏平台 issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.
European Medicines Agency Validates 澳门正规博彩十大排行平台’s Marketing Authorization Application for Vosoritide to Treat Children with Achondroplasia
澳门正规博彩十大排行平台 announced in August that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for vosoritide.
澳门正规博彩十大排行平台 Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia
澳门正规博彩十大排行平台 announced in July that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vosoritide.
澳门正规博彩十大排行平台 Promotes Company Veteran, Brian R. Mueller, to Executive Vice President, CFO
澳门正规博彩十大排行平台 announced in June the promotions of Brian R. Mueller to Executive Vice President, Chief Financial Officer and of Andrea L. Acosta to Group Vice President, Chief Accounting Officer.
澳门正规博彩十大排行平台 Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit
澳门正规博彩十大排行平台 announced in June additional data from its previously reported four-year update of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for severe hemophilia A.
澳门正规博彩十大排行平台 Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
澳门正规博彩十大排行平台 announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A.
澳门正规博彩十大排行平台 Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic Cardiomyopathies
澳门正规博彩十大排行平台 announced in May that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies.
澳门正规博彩十大排行平台 Appoints Global Pharmaceutical Veteran C. Greg Guyer, Ph.D., to Chief Technical Officer, Executive Vice President of Global Manufacturing and Technical Operations, Effective May 4
澳门正规博彩十大排行平台 announced in April the appointment of C. Greg Guyer, Ph.D., to Chief Technical Officer, Executive Vice President of Global Manufacturing and Technical Operations.
澳门正规博彩十大排行平台 Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe
澳门正规博彩十大排行平台 announced in April that based on recent meetings with health authorities in the US and Europe, the Company plans to submit marketing applications to the US Food and Drug Administration (澳门十大博彩正规游戏平台) and the European Medicines Agency (EMA) in the third quarter of 2020 for vosoritide.
澳门正规博彩十大排行平台’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by 澳门十大博彩正规游戏平台 with Review Action Date of August 21, 2020
澳门正规博彩十大排行平台 announced in February that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) has accepted for Priority Review the Biologics License Application (BLA) to the 澳门十大博彩正规游戏平台 for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. This acceptance by the 澳门十大博彩正规游戏平台 marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the United States.
澳门正规博彩十大排行平台, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy
澳门正规博彩十大排行平台 announced in January that both the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.
澳门正规博彩十大排行平台 Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
澳门正规博彩十大排行平台 announced in January that the New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe
2019
澳门正规博彩十大排行平台 Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years
澳门正规博彩十大排行平台 announced in February that an ongoing open-label extension study treating patients with Brineura® (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort of CLN2 disease for three years as measured by the CLN2 Clinical Rating Scale.
澳门正规博彩十大排行平台 Receives Positive CHMP Opinion in Europe for Palynziq® (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older
澳门正规博彩十大排行平台 announced in March that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Palynziq® (pegvaliase) Injection to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. In addition, the CHMP noted that the data collected in the Phase 3 trial and extension study was suggestive of an improvement in inattention and mood symptoms.
Forbes Names 澳门正规博彩十大排行平台 4th Best Midsize Employer in America
澳门正规博彩十大排行平台 announced in April that it has been ranked fourth on Forbes magazine’s 2019 list of “America’s Best Midsize Employers,” increasing from 51st on last year’s 2018 list. 澳门正规博彩十大排行平台 was ranked first among its peers in the ‘Drugs & Biotechnology’ industry.
European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older
澳门正规博彩十大排行平台 announced in May that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options.
澳门正规博彩十大排行平台 Provides 3 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
澳门正规博彩十大排行平台 announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec. The three-year update demonstrated that bleed rate control with valoctocogene roxaparvovec 6e13 vg/kg dose was maintained for a third year with a median Annualized Bleed Rate (ABR) of 0 and mean ABR of 0.7 in that year. In addition, Factor VIII levels in the 6e13 vg/kg dose appeared to be approaching a plateau in year three. Factor VIII levels measured with the chromogenic substrate (CS) assay at the end of year three were mean and median of 32.7 IU/dL and 19.9 IU/dl, respectively, compared with mean and median of 36.4 IU/dL and 26.2 IU/dL, respectively, at the end of year two.
澳门正规博彩十大排行平台 Announces that Phase 3 Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe
澳门正规博彩十大排行平台 announced in May that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe.
澳门正规博彩十大排行平台 Announces Approval of Vimizim® (elosulfase alfa) in China for Treatment of Morquio A Syndrome
澳门正规博彩十大排行平台 announced in June that Vimizim® (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.
澳门正规博彩十大排行平台 Announces New England Journal of Medicine Publishes Vosoritide Phase 2 Study Showing Sustained Annualized Growth Up to 42 Months in Children with Achondroplasia
澳门正规博彩十大排行平台 announced in June that the New England Journal of Medicine (NEJM) published online results from a Phase 2 dose-finding and extension study for vosoritide.
澳门正规博彩十大排行平台 Earns Milestone Payments from Pfizer for TALZENNA® (talazoparib) for Metastatic Breast Cancer Patients with an Inherited BRCA Mutation
澳门正规博彩十大排行平台 Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Company earned a $15 million milestone payment from Pfizer, Inc. This milestone payment was triggered by the European Commission (EC) approval of TALZENNA® (talazoparib) as monotherapy for the treatment of adult patients with germline breast cancer susceptibility gene (gBRCA) 1/2-mutations, who have human epidermal growth factor receptor 2-negative (HER2-) locally advanced (LA) or metastatic breast cancer (MBC).
澳门正规博彩十大排行平台 Plans Regulatory Submissions for Marketing Authorization of Valoctocogene Roxaparvovec to Treat Severe Hemophilia A in 4Q 2019 in both U.S. and Europe
澳门正规博彩十大排行平台 announced in July that based on recent meetings with health authorities in the U.S. and Europe, the company plans to submit marketing applications to both the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) and the European Medicines Agency (EMA) in 4Q 2019 for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
澳门正规博彩十大排行平台 Appoints Pharmaceutical Veteran and Former J & J Executive, Liz McKee Anderson, to Board of Directors
澳门正规博彩十大排行平台 announced in July the appointment of pharmaceutical veteran and former Johnson & Johnson executive, Liz McKee Anderson, to its Board of Directors effective July 15th, 2019. Ms. Anderson serves on the boards of a number of life sciences companies. Before retiring, Ms. Anderson spent 11 years in executive roles of increasing responsibility at Johnson & Johnson.
澳门正规博彩十大排行平台, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in U.K. for Investigational Gene Therapy for PKU
澳门正规博彩十大排行平台 announced in September that it had submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to reduce blood phenylalanine (Phe) concentrations levels in patients with PKU.
澳门正规博彩十大排行平台 Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study
澳门正规博彩十大排行平台 provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of disproportionate short stature in humans, at its annual R&D Day.
澳门正规博彩十大排行平台 Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
澳门正规博彩十大排行平台 announced in November that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
澳门正规博彩十大排行平台 Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide
澳门正规博彩十大排行平台 reported in December positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr (p<0.0001). The results were consistent across the broad patient population studied. Vosoritide was generally well tolerated with no clinically significant blood pressure decreases.
European Medicines Agency Validates 澳门正规博彩十大排行平台’s Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
澳门正规博彩十大排行平台 announced in December that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
澳门正规博彩十大排行平台 Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A
澳门正规博彩十大排行平台 announced in December that the company had submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
2018
WORLDSymposium Recognizes 澳门正规博彩十大排行平台’s Brineura® (cerliponase alfa) with 2018 New Treatment Award
澳门正规博彩十大排行平台 announced in February that The WORLDSymposium 2018 awarded Brineura® (cerliponase alfa) the New Treatment Award, which recognizes important achievements in therapeutic advancements for lysosomal diseases. The award was presented on Monday, February 5.
澳门正规博彩十大排行平台 Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium™ 2018
澳门正规博彩十大排行平台 announced in February that it presented interim data from a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB) at WORLDSymposium™ 2018.
Rare Disease Day 2018: 澳门正规博彩十大排行平台 Launches RARE Scholars Scholarship Program
澳门正规博彩十大排行平台 announced in February the launch of RARE Scholars, an annual scholarship for students living with rare disease. RARE Scholars aims to empower patients with continued education by recognizing students living with rare diseases who have demonstrated leadership and participation in school and community activities. The RARE Scholars program will distribute up to $20,000 each year in June, awarding $5,000 for undergraduate four-year or graduate studies or $2,500 for two-year or vocational-technical studies.
澳门正规博彩十大排行平台’s Gene Therapy Manufacturing Facility Recognized with Industry Award
澳门正规博彩十大排行平台 announced in March that the International Society for Pharmaceutical Engineering (ISPE) selected the company’s gene therapy manufacturing facility as the 2018 Facility of the Year Category Winner for Project Execution. The recognition highlighted the company’s successful construction of the facility in Novato, CA, which took less than a year to transform basic infrastructure into one of the first gene manufacturing facilities of its kind in the world. Winners were announced on Tuesday, March 20 during the 2018 Europe Annual Conference in Rome, Italy.
European Medicines Agency (EMA) Accepts 澳门正规博彩十大排行平台’s Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU)
澳门正规博彩十大排行平台 announced in March that the European Medicines Agency (EMA) has accepted 澳门正规博彩十大排行平台’s submission of a Marketing Authorization Application (MAA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options including sapropterin.
New England Journal of Medicine Published Open-label Study Showing Brineura® (cerliponase alfa) Reduced the Rate of Clinical Decline of Children with CLN2 Disease, a Form of Batten Disease
澳门正规博彩十大排行平台 announced in April that the New England Journal of Medicine (NEJM) published updated results from a multi-center, open-label, dose-escalation and ongoing extension study evaluating the efficacy and safety of Brineura® (cerliponase alfa) in children with CLN2 disease in the May 2018 issue. The new data demonstrated that treatment with Brineura resulted in less decline in motor and language function compared to historical controls.
澳门正规博彩十大排行平台 Named To Forbes List Of America’s Best Mid-size Employers
澳门正规博彩十大排行平台 announced in May that it had been ranked 51st overall among 500 companies on Forbes magazine’s 2018 list of “America’s Best Mid-size Employers,” and third among companies in the biotechnology industry.
澳门正规博彩十大排行平台 Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies
澳门正规博彩十大排行平台 announced in May that it had dosed the first patient in a Phase 1/2 study (BMN 270-203) evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.
澳门正规博彩十大排行平台 Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress
澳门正规博彩十大排行平台 announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during an oral presentation at the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, Scotland by John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry and primary investigator for this Phase 1/2 study.
澳门正规博彩十大排行平台 Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease
澳门正规博彩十大排行平台 announced in May that it had received standard approval from the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for Palynziq™ (pegvaliase-pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is 澳门正规博彩十大排行平台’s second approved treatment for this important condition.
澳门正规博彩十大排行平台 Receives Milestone Payments from Pfizer for Talazoparib
澳门正规博彩十大排行平台 announced in June that the Company received $20 million in milestone payments from Pfizer Inc. These milestone payments were triggered by the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) acceptance of Pfizer’s New Drug Application (NDA) submission for talazoparib and by the European Medicines Agency (EMA) acceptance of Pfizer’s submission of a Marketing Authorization Application (MAA) for talazoparib. These milestone payments are part of an agreement made with Medivation, Inc. when the company purchased talazoparib. Medivation was acquired by Pfizer.
澳门正规博彩十大排行平台 Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
澳门正规博彩十大排行平台 announced in June that the company dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia, the most common form of disproportionate short stature in humans.
澳门正规博彩十大排行平台 Announces First Recipients of RARE Scholars Scholarship Program
澳门正规博彩十大排行平台 announced in June that it has awarded the first five recipients of the RARE Scholars program, an annual scholarship for students living with rare disease. The RARE Scholars program provides up to $20,000 to students living with mucopolysaccharidoses, phenylketonuria or Batten disease.
澳门正规博彩十大排行平台 Partners with Believe Limited for ‘Breaking Through!’ Musical Theater Intensive
澳门正规博彩十大排行平台 announced an exclusive partnership with Believe Limited to produce the ‘Breaking Through!’ musical theater intensive. The first-of-its-kind program is a three-day music workshop for the bleeding disorders community and is designed to provide young adults with powerful education on the healing and therapeutic power of the arts and self-expression. The musical workshop, directed by hemophilia advocate and Believe Limited CEO Patrick James Lynch, will be held from November 9 – November 12, 2018 and will culminate in a Broadway-style performance for local bleeding disorder community members, family and friends.
Forbes Names 澳门正规博彩十大排行平台 Among World’s Best Employers
澳门正规博彩十大排行平台 announced in October that Forbes’ named the company to its list, Global 2000: World’s Best Employers. It is ranked within the top 300 out of a total of 2000 companies and fifth among the nine biotech companies also named. This marks the second time this year that 澳门正规博彩十大排行平台 has been acknowledged as a top place to work by Forbes, having also been named to the magazine’s list of Best Mid-Sized Employers, earlier this year with a ranking of 51 among 500 companies and third among biotech companies also named.
澳门正规博彩十大排行平台 and Believe Limited Announce Selection of 25 High School Students to Perform in First-of-its-Kind ‘Hemophilia: The Musical’
澳门正规博彩十大排行平台 announced in October the twenty-five high school students from across the U.S. who will participate in ‘Hemophilia: The Musical,’ a first-of-its-kind theatrical production written and informed by their unique experiences of life as a young person with a bleeding disorder. The musical will take place at New World Stages on November 12, 2018 at 1 pm ET. A live stream of the performance will be made available at BreakingThroughHemophilia.com.
澳门正规博彩十大排行平台 Receives Milestone Payments from Pfizer for Talzenna® (Talazoparib) for Metastatic Breast Cancer Patients with an Inherited BRCA Mutation
澳门正规博彩十大排行平台 announced in October that the Company earned $15 million in milestone payments from Pfizer Inc. These milestone payments were triggered by the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) approval of Talzenna® (talazoparib) for the treatment of adult patients with deleterious or suspected deleterious germline BRCA (gBRCA)-mutated, HER2-negative locally advanced (LA) or metastatic breast cancer (MBC). Patients are selected for therapy based on an 澳门十大博彩正规游戏平台-approved companion diagnostic.
澳门正规博彩十大排行平台 and Believe Limited Announce the Debut of ‘Hemophilia: The Musical’
澳门正规博彩十大排行平台 announced in November the debut of the Broadway-style ‘Hemophilia: The Musical,’ a first-of-its-kind theatrical production featuring 25 students affected by a bleeding disorder. A recording of the six-song performance is now available at BreakingThroughHemophilia.com.
2017
澳门正规博彩十大排行平台 Receives Access to Priority Medicines (PRIME) Regulatory Support from EMA for BMN 270 Gene Therapy in Hemophilia A
澳门正规博彩十大排行平台 announced in February that the European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) regulatory initiative for the company’s investigational gene therapy treatment for severe hemophilia A, BMN 270. To be accepted for PRIME, an investigational therapy has to show its potential to benefit patients with unmet medical needs based on early clinical data.
澳门正规博彩十大排行平台 Announces Kuvan® (sapropterin dihydrochloride) Patent Challenge Settlement
澳门正规博彩十大排行平台 announced in April that it has entered into a settlement agreement with Par Pharmaceutical that resolves patent litigation in the United States (U.S.) related to 澳门正规博彩十大排行平台’s Kuvan® (sapropterin dihydrochloride) 100mg oral tablets and powder for oral solution in 100mg packets.
澳门正规博彩十大排行平台 Receives Positive CHMP Opinion in Europe for Brineura™ (cerliponase alfa) for First Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare and Fatal Brain Disorder in Children
澳门正规博彩十大排行平台 announced in April that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 (TPP1) deficiency.
澳门十大博彩正规游戏平台 Approves Brineura™ (cerliponase alfa) for the Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare Pediatric Brain Disorder in Children
澳门正规博彩十大排行平台 announced in April that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) approved Brineura™ (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.
European Commission Approves Brineura™ (cerliponase alfa), the First Treatment for CLN2 Disease, a Form of Batten Disease and Ultra-Rare Brain Disorder in Children
澳门正规博彩十大排行平台 announced in June that the European Commission (EC) has granted marketing authorization for Brineura™ (cerliponase alfa), the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. The dosing administration includes all ages from birth.
澳门正规博彩十大排行平台 Submits Pegvaliase Biologics License Application (BLA) to the U.S. 澳门十大博彩正规游戏平台 for Treatment of Phenylketonuria (PKU)
澳门正规博彩十大排行平台 announced that the company submitted a Biologics License Application (BLA) on Friday, June 30, 2017 to the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with PKU who have uncontrolled blood Phe levels on existing management.
澳门正规博彩十大排行平台’s Investigational Gene Therapy for Hemophilia A at 6e13 vg/kg Dose Maintains Average Factor VIII Levels within Normal Range for over One Year
澳门正规博彩十大排行平台 announced in July an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A.
Sarepta Therapeutics and 澳门正规博彩十大排行平台 Pharmaceutical Inc. Announce Execution of a Global Settlement and a License Agreement Resolving Exon Skipping Patent Litigation
Sarepta Therapeutics and 澳门正规博彩十大排行平台 announced in July that they had executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to 澳门正规博彩十大排行平台’s DMD patent estate for EXONDYS 51 and all future exon-skipping products. 澳门正规博彩十大排行平台 retains the right to convert the license to a co-exclusive right in the event it decides to proceed with an exon-skipping therapy for DMD. In addition, Sarepta and 澳门正规博彩十大排行平台 executed a settlement agreement, resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD.
澳门正规博彩十大排行平台 Announces Plans to Progress Both the 6e13vg/kg and 4e13 vg/kg Doses of BMN 270, its Investigational Gene Therapy for Hemophilia A, into Phase 3 Studies
澳门正规博彩十大排行平台 announced in August that it will expand its development plan for BMN 270, its investigational gene therapy for hemophilia A, to include an additional Phase 3 study of the 4e13 vg/kg dose based on updated data as of July 28, 2017 from its ongoing open-label Phase 1/2 study of BMN 270.
Forbes Ranks 澳门正规博彩十大排行平台 12th Most Innovative Company in the World
澳门正规博彩十大排行平台 announced in August that it has been ranked 12th on Forbes magazine’s 2017 list of the “World’s Most Innovative Companies.”
澳门十大博彩正规游戏平台 Accepts 澳门正规博彩十大排行平台’s Pegvaliase Biologics License Application (BLA) and Grants Priority Review Designation
澳门正规博彩十大排行平台 announced in August that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) has accepted for Priority Review the Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management.
澳门正规博彩十大排行平台 Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017
澳门正规博彩十大排行平台 announced in September that it presented interim data from the dose escalation arm of a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB) at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017.
澳门正规博彩十大排行平台 Appoints Robert J. Hombach, Former Baxalta CFO and COO, to Board of Directors
澳门正规博彩十大排行平台 announced in October the appointment of former Baxalta Chief Financial Officer and Chief Operations Officer, Robert J. Hombach, to its Board of Directors.
Popular Science Names 澳门正规博彩十大排行平台’s Brineura® (cerliponase alfa) One of the Top Health Innovations of 2017 with “Best of What’s New” Award
澳门正规博彩十大排行平台 announced in October that Brineura® (cerliponase alfa) has been awarded the 2017 Popular Science “Best of What’s New” award in the health category. Each year, Popular Science reviews thousands of new products and innovations across 11 categories for its annual “Best of What’s New” issue, selecting those that represent a significant step forward in their category.
澳门十大博彩正规游戏平台 Grants Breakthrough Therapy Designation for 澳门正规博彩十大排行平台’s Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A
澳门正规博彩十大排行平台 announced in October that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation.
澳门正规博彩十大排行平台 Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Publication
澳门正规博彩十大排行平台 announced in December an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A.
澳门正规博彩十大排行平台 Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting
澳门正规博彩十大排行平台 announced in December updates on valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia at ASH.
澳门正规博彩十大排行平台 Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
澳门正规博彩十大排行平台 announced in December that it has dosed the first patient in the global GENEr8-1 Phase 3 study with the 6e13 vg/kg dose for valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy for the treatment of patients with severe hemophilia A.
澳门正规博彩十大排行平台 Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018
澳门正规博彩十大排行平台 announced in December that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) will require additional time to complete its review of the Biologics License Application (BLA) for its investigational therapy pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management.
2016
澳门正规博彩十大排行平台 Announces Addition of David Pyott, Former Allergan Chairman and CEO, to Company’s Board of Directors
澳门正规博彩十大排行平台 announced in January that David Pyott had been elected to the company’s board of directors. Mr. Pyott is the former Chairman and Chief Executive Officer of Allergan and joined the 澳门正规博彩十大排行平台 Board immediately.
澳门正规博彩十大排行平台 Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference
澳门正规博彩十大排行平台 announced in January interim results from INSPIRE, a Phase 2 trial for reveglucosidase alfa, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) being studied for the treatment of late-onset Pompe disease (LOPD). The interim efficacy and safety analysis is based on 24 patients who previously had been on treatment with the enzyme replacement therapy, alglucosidase alfa, and were switched to reveglucosidase alfa.
澳门十大博彩正规游戏平台 Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
澳门正规博彩十大排行平台 announced in January that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) issued a Complete Response letter to the Company’s New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.
澳门正规博彩十大排行平台 Announces 澳门正规博彩十大排行平台 RareConnections™ Rebranding and Consolidating 澳门正规博彩十大排行平台’s Comprehensive Support and Services for Eligible Rare Disease Patients
澳门正规博彩十大排行平台 announced in February the launch of 澳门正规博彩十大排行平台 RareConnectionsTM, rebranding its patient-focused offering formally known as the 澳门正规博彩十大排行平台 Patient and Physician Support (BPPS). For over 10 years, 澳门正规博彩十大排行平台 has been a leader in designing and implementing multiple separate service offerings to support our patients. With the launch of 澳门正规博彩十大排行平台 RareConnections, it now provides them under one expanded program, which includes support for eligible patients throughout treatment and assistance to gain and maintain access to 澳门正规博彩十大排行平台’s innovative therapies.
澳门正规博彩十大排行平台 Receives Orphan Drug Designation From 澳门十大博彩正规游戏平台 for First AAV-Factor VIII Gene Therapy, BMN 270, for Patients With Hemophilia A
澳门正规博彩十大排行平台 announced in March that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台). 澳门正规博彩十大排行平台 is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April. BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. The 澳门十大博彩正规游戏平台 Orphan Drug program provides orphan designation to drugs and biologics that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States).
澳门正规博彩十大排行平台 Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium(TM) 2016
澳门正规博彩十大排行平台 announced in March positive 48-week results from its Phase 1/2 pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease. The average rate of clinical decline for motor and language function in patients receiving cerliponase alfa treatment — the primary efficacy endpoint — was approximately 80% less than the expected rate of decline in the untreated population, preserving essential function in the majority of treated patients (p <0.0001). Treatment with 300 mg cerliponase alfa administered via intracerebroventricular (ICV) infusion every other week was generally safe and well-tolerated in 24 patients and resulted in disease stabilization in 65% (15 of 23) of patients treated over a 48-week period, based on the Hamburg Motor + Language CLN2 rating. 澳门正规博彩十大排行平台 estimates the incidence of CLN2 disease is approximately one in 200,000 with approximately 1,200 to 1,600 children in 澳门正规博彩十大排行平台's commercial territories.
澳门正规博彩十大排行平台 Phase 3 Study of Pegvaliase for Phenylketonuria (PKU) Meets Primary Endpoint of Blood Phenylalanine (Phe) Reduction (p<0.0001)
澳门正规博彩十大排行平台 announced in March that the pivotal Phase 3 PRISM-2 study (formerly referred to as 165-302) of pegvaliase met the primary endpoint of change in blood Phe compared with placebo (p<0.0001) in preliminary results. During the 8 week PRISM-2 double-blind, placebo-controlled, randomized drug discontinuation trial (RDT), 86 patients were randomized to either remain on pegvaliase or receive matching placebo. The pegvaliase treated group maintained mean blood Phe levels at 527.2 umol/L compared to their RDT baseline of 503.9 umol/L, whereas the placebo treated group mean blood Phe levels increased to 1385.7 umol/L compared to their RDT baseline of 536.0 umol/L . (see Table 1) The treatment effect demonstrated in this study represents an approximately 62% improvement in blood Phe compared to placebo.
澳门正规博彩十大排行平台 Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patients with Hemophilia A
澳门正规博彩十大排行平台 announced in March that BMN 270, an investigational gene therapy for the treatment of hemophilia A, has been granted orphan drug designation by the European Commission. In the European Union, orphan drug designation is given to treatments that are intended for life-threatening or chronically-debilitating conditions with a prevalence of not more than 5 in 10,000 people. Earlier this month, 澳门正规博彩十大排行平台 announced BMN 270 had also received orphan drug designation from the U.S. Food and Drug Administration.
澳门正规博彩十大排行平台 Provides Encouraging Preliminary Data on First 8 Patients in Hemophilia A Gene Therapy Program
澳门正规博彩十大排行平台 announced in April preliminary data from an ongoing Phase 1/2 clinical trial with BMN 270, an investigational gene therapy treatment for hemophilia A. A total of eight patients with severe hemophilia A received a single dose of BMN 270, six of whom have been treated at the highest dose of 6 x 1013 vg/kg, and to date, post-treatment follow-up ranges from five to 16 weeks. At last observation, patients at the highest dose experienced increasing Factor VIII activity levels ranging between 4% and 60% (as a percentage calculated based on the numbers of International Units (IU) per milliliter of whole blood), with five of six patients treated at the high dose now over 5% and two of six at over 50%. All high dose patients improved from severe to either moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.
澳门正规博彩十大排行平台 Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)
澳门正规博彩十大排行平台 announced in April that it had enrolled the first patient in a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB). Discovered by 澳门正规博彩十大排行平台, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with MPS IIIB receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via intracerebroventricular (ICV) infusion.
澳门正规博彩十大排行平台 Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease
澳门正规博彩十大排行平台 announced in May that the European Medicines Agency (EMA) has granted 澳门正规博彩十大排行平台’s request for accelerated assessment for the planned cerliponase alfa Marketing Authorization Application (MAA). Accelerated assessments are granted on the grounds that a product may satisfy an unmet medical need and is of major interest from the point of view of therapeutic innovation and public health. Accelerated assessment has the potential to shorten EMA’s review procedure. However, at any time during the MAA assessment, the EMA may decide to continue the assessment under standard assessment timelines, and most applications that initially qualify for accelerated assessment are ultimately reviewed on a standard timeline.
澳门正规博彩十大排行平台 Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe
澳门正规博彩十大排行平台 announced in May that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
澳门正规博彩十大排行平台 Appoints Two BioPharmaceutical Veterans to Board of Directors, Willard Dere, M.D. and Kathryn E. Falberg
澳门正规博彩十大排行平台 announced in July the appointment of two biopharmaceutical veterans to its Board of Directors: Willard Dere, M.D., Professor of Internal Medicine at the University of Utah Health Sciences Center and former Senior Vice President in Research and Development at Amgen and Kathryn E. Falberg, former Chief Financial Officer of Jazz Pharmaceuticals and Amgen.
澳门正规博彩十大排行平台 Provides Positive Proof-of-Concept Data for BMN 270 Gene Therapy in Hemophilia A in Late Breaking Oral Presentation at the World Federation of Hemophilia (WFH) 2016 World Congress
澳门正规博彩十大排行平台 announced in July positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A at the XXXII International Congress of the World Federation of Hemophilia (WFH). The data was presented in the Late Breaking Gene Therapy session by John Pasi, Professor of Haemostasis and Thrombosis, Barts and the London School of Medicine, Honorary Consultant Haematologist, The Royal London Hospital, and a lead investigator of the study.
澳门十大博彩正规游戏平台 Accepts BLA for 澳门正规博彩十大排行平台’s Cerliponase Alfa for CLN2 Disease, Form of Batten Disease
澳门正规博彩十大排行平台 announced in July that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa, an investigational therapy to treat children with CLN2 disease, a form of Batten disease. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is January 27, 2017. 澳门正规博彩十大排行平台 also has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for cerliponase alfa, and it is undergoing validation at the Agency.
澳门正规博彩十大排行平台 Announces Pricing of Public Offering of Common Stock
澳门正规博彩十大排行平台 announced in August the pricing of an underwritten public offering of 7,500,000 shares of its common stock at a price to the public of $96.00 per share. The gross proceeds to 澳门正规博彩十大排行平台 from this offering are expected to be approximately $720.0 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by 澳门正规博彩十大排行平台.
Forbes Ranks 澳门正规博彩十大排行平台 10th Most Innovative Company in the World
澳门正规博彩十大排行平台 announced in August that it has been ranked 10th on Forbes magazine’s 2016 list of the “World’s Most Innovative Companies.” This is the third consecutive year that 澳门正规博彩十大排行平台 has received the recognition, acknowledging the company’s ongoing commitment to rapidly developing and delivering first-in-class or best-in-class therapies for those with severe or life-threatening rare diseases. Last year, the company was also ranked 10th, and in 2014, the company ranked 7th.
澳门正规博彩十大排行平台 Announces Update to Brineura™ (Cerliponase Alfa) Program for Treatment of CLN2 Disease, a Form of Batten Disease
澳门正规博彩十大排行平台 announced in September a program update for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease.
澳门正规博彩十大排行平台 Announces EMA Validation of Brineura™ (Cerliponase Alfa) Marketing Authorization Application for Treatment of CLN2 Disease, a Form of Batten Disease
澳门正规博彩十大排行平台 announced in September that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP).
澳门正规博彩十大排行平台 Reviews Status of Exon 51 Composition of Matter and Method of Use Patent Interference Cases against Sarepta Therapeutics
澳门正规博彩十大排行平台 announced in September that it intends to seek a review of the Patent Trial and Appeal Board (PTAB) of the United States ruling in Interference No. 106,008, related to composition of matter (COM) claims related to exon 51 skipping antisense oligonucleotides. 澳门正规博彩十大排行平台 is completing its review of the decision and the specific means it may use to seek a further review.
UK Regulatory Agency Approves Continued Enrollment in 澳门正规博彩十大排行平台 Phase 1/2 Study of BMN 270 in Hemophilia A
澳门正规博彩十大排行平台 announced in October that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A.
澳门正规博彩十大排行平台 Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics (ASHG) 2016 Meeting
澳门正规博彩十大排行平台 provided an update on its Phase 2 study of vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from 8 children in cohort 4, who completed six months of daily dosing at 30 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). These data are comparable to those observed at the lower dose of 15 µg/kg/day in cohort 3. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 µg/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.01).
澳门正规博彩十大排行平台 Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia
澳门正规博彩十大排行平台 announced in December that the company has initiated a global Phase 3 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism. The first child enrolled in the study was at a site in Australia.
2015
Ernst & Young Names 澳门正规博彩十大排行平台 CEO Jean-Jacques Bienaime EY Entrepreneur Of The Year® 2015 National Award Winner in Life Sciences
Ernst & Young Global Limited in November awarded 澳门正规博彩十大排行平台 CEO Jean-Jacques Bienaimé the EY Entrepreneur Of The Year® 2015 National Award in the Life Sciences category. The award recognizes outstanding entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance, and personal commitment to their businesses and communities.
澳门正规博彩十大排行平台’s Initial 6-Month Data from Phase 2 Study of Vosoritide (BMN 111) in Children with Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting
Dr. Melita Irving, Clinical Geneticist, Guy’s and St Thomas’ NHS Foundation Trust, Evelina Children’s Hospital London, UK, in October presented the initial six-month data from the first three cohorts of 澳门正规博彩十大排行平台’s Phase 2 proof-of-concept and dose-finding study of vosoritide (BMN 111), an analog of C-type Natriuretic Peptide, in children with achondroplasia at the American Society for Bone and Mineral Research Annual 2015 Meeting in Seattle, Washington. Achondroplasia is the most common form of human dwarfism. The initial six-month data from the first three cohorts showed a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (p-value= 0.01).
澳门正规博彩十大排行平台 to Acquire Rights to Phenylketonuria (PKU) Franchise From Merck Serono
澳门正规博彩十大排行平台 in October announced that it acquired all global rights to Kuvan® (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Under the terms of the agreement, 澳门正规博彩十大排行平台 will provide Merck with an upfront payment of €340 million. An additional €60 million in milestones will be paid to Merck if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds. In addition, €125 million will be paid to Merck for regulatory milestones related to pegvaliase. 澳门正规博彩十大排行平台 will now have exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan.
澳门正规博彩十大排行平台 Enrolls First Patient in Phase 1/2 Trial of Gene Therapy Drug Candidate BMN 270 for the Treatment of Hemophilia A
澳门正规博彩十大排行平台 in September announced that it has enrolled the first patient in a Phase 1/2 trial for BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A.
Forbes Ranks 澳门正规博彩十大排行平台 10th Most Innovative Company in the World
Forbes magazine in August ranked 澳门正规博彩十大排行平台 10th on its 2015 list of the “World’s Most Innovative Companies.” This is the second consecutive year that 澳门正规博彩十大排行平台 has received the recognition, reinforcing the company’s promise to deliver new therapeutics to patients with severe or life-threatening diseases. The company was previously ranked seventh.
澳门正规博彩十大排行平台 Joins the NASDAQ-100 Index
澳门正规博彩十大排行平台 in July announced that it will become a component of the NASDAQ-100 Index® (NASDAQ:NDX), the NASDAQ-100 Equal Weighted Index (NASDAQ:NDXE) and the NASDAQ-100 Ex-Technology Index (NASDAQ:NDXX) prior to market open on Monday, July 27, 2015. The NASDAQ-100 Index is composed of the 100 largest non-financial stocks listed on The NASDAQ Stock Market based on market capitalization.
澳门正规博彩十大排行平台 Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The European Medicines Agency (EMA) in June validated the Marketing Authorization Application (MAA) for 澳门正规博彩十大排行平台’s drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA’s standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.
BMN 111 (vosoritide) Improves Growth Velocity in Children With Achondroplasia in Phase 2 Study
澳门正规博彩十大排行平台 in June announced positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia. Achondroplasia is the most common form of human dwarfism. Vosoritide has Orphan designation in both the United States and Europe.
澳门正规博彩十大排行平台 CEO Jean-Jacques Bienaime Awarded EY Entrepreneur Of The Year(R) 2015 Award in Northern California in Health and Life Sciences
EY in June awarded 澳门正规博彩十大排行平台 CEO Jean-Jacques Bienaimé the EY Entrepreneur Of The Year® 2015 Award in the Health and Life Sciences category in Northern California. The award recognizes outstanding entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance, and personal commitment to their businesses and communities.
澳门正规博彩十大排行平台 Announces Completion of Tender Offer for Prosensa’s Shares and Commences Subsequent Offering Period
澳门正规博彩十大排行平台 in January announced it completed the initial offering period for the previously announced tender offer for all of the outstanding ordinary shares of Prosensa Holding N.V. (Nasdaq:RNA). As a result, 澳门正规博彩十大排行平台 will purchase approximately 93.4% of Prosensa’s outstanding shares pursuant to the initial offering period for the tender offer. 澳门正规博彩十大排行平台 also announced that it has commenced a subsequent offering period to provide Prosensa shareholders who have not yet tendered their shares the opportunity to do so. The subsequent offering period is scheduled to expire at 6:00 p.m., New York City time, on January 29, 2015.
澳门正规博彩十大排行平台 Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease
澳门正规博彩十大排行平台 in January announced interim results from its Phase 1/2 pivotal study for BMN 190 or cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), to treat of patients with late infantile CLN2 disease, a form of Batten disease. Interim data indicates that in all nine of the BMN 190 patients who have been followed for at least six months and up to 15 months, the treatment appears to show stabilization of the disease compared to the natural history based on a standardized measure of motor and language function.
2014
澳门正规博彩十大排行平台 Announces Approval of Vimizim® (elosulfase alfa) in Japan for Treatment of Morquio A Syndrome
Japan’s Ministry of Health, Labor and Welfare granted approval of the registration of 澳门正规博彩十大排行平台’s Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Japan approved for this condition. Vimizim was reviewed under the Orphan Drug program.
澳门正规博彩十大排行平台’s Vimizim® (elosulfase alfa) Approved in Brazil for Treatment of Morquio A Syndrome
The Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil, in December approved 澳门正规博彩十大排行平台’s Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first and only treatment for the ultra-rare genetic condition.
澳门十大博彩正规游戏平台 Grants 澳门正规博彩十大排行平台 Orphan Drug Designation for NAGLU Fusion Protein, Tralesinidase alfa (BMN 250), for MPS IIIB
The Food and Drug Administration in December granted orphan drug designation for Tralesinidase alfa (BMN 250), a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo Syndrome Type B or Mucopolysaccharidosis IIIB (MPS IIIB). Tralesinidase alfa (BMN 250) is an enzyme replacement therapy using recombinant human NAGLU with an IGF2, or Glycosylation Independent Lysosomal Targeting (GILT) tag. Tralesinidase alfa (BMRN 250) is delivered directly to the brain using 澳门正规博彩十大排行平台’s patented technology.
澳门正规博彩十大排行平台’s Vimizim® (elosulfase alfa) Approved in Australia for Treatment of Morquio A Syndrome
In December, the Australian Therapeutic Goods Administration granted approval of the registration of Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Australia approved for this condition.
澳门正规博彩十大排行平台 Agrees to Acquire Prosensa Holding N.V.
In November, 澳门正规博彩十大排行平台 entered a definitive agreement to offer to purchase all of the outstanding ordinary shares of Prosensa Holding N.V. for $17.75 per share, for a total up front consideration of approximately $680 million. In addition, two approximately $80 million contingent milestones are payable for the approval of drisapersen, Prosensa’s drug candidate in development for certain forms of Duchenne muscular dystrophy in the U.S. no later than May 15, 2016 and Europe no later than February 15, 2017, respectively.
澳门正规博彩十大排行平台 Named by CenterWatch as One of the Fastest Developers of Medicines
In September, CenterWatch, a leading source for global clinical trial information, named 澳门正规博彩十大排行平台 as one of the fastest drug developers in the industry.
澳门正规博彩十大排行平台 Sells Priority Review Voucher for $67.5 Million
In July, 澳门正规博彩十大排行平台 announced that it has sold the Rare Pediatric Disease Priority Review Voucher it obtained when it won approval of Vimizim in February under an 澳门十大博彩正规游戏平台 program intended to encourage the development of treatments for rare pediatric diseases, for $67.5 million to Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc.
澳门正规博彩十大排行平台 Announces Health Canada Approval of Vimizim™ (elosulfase alfa) for the Treatment of Morquio A Syndrome
Health Canada in July approved Vimizim® (elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes Vimizim the first and only pharmaceutical treatment option available in Canada for children and adults living with this severely debilitating, progressive and life-limiting disorder.
澳门正规博彩十大排行平台 Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease
澳门正规博彩十大排行平台 in May announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe disease. BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), designed to target delivery to the lysosomes where the enzyme is most needed.
澳门正规博彩十大排行平台 Announces European Commission Approval for Vimizim(R) (elosulfase alfa) for the Treatment of Morquio A Syndrome in Patients of All Ages
The European Commission in April granted marketing authorization for Vimizim® (elosulfase alfa), the first specific treatment approved in the European Union for Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, in patients of all ages. As the first drug ever approved for Morquio A syndrome, Vimizim has been granted orphan drug status in the European Union, which confers ten years of market exclusivity.
澳门正规博彩十大排行平台 Appoints Pioneer in Personalized Medicine, Dennis J. Slamon, M.D., Ph.D., to Board of Directors
澳门正规博彩十大排行平台 in March announced the appointment of Dennis J. Slamon, M.D., Ph.D., director of Clinical/Translational Research and director of the Revlon/UCLA Women’s Cancer Research Program at UCLA’s Jonsson Comprehensive Cancer Center, to the company’s board of directors.
澳门正规博彩十大排行平台 Announces Pricing of Public Offering of Common Stock
澳门正规博彩十大排行平台 announced today the pricing on March 4, 2014 of an underwritten public offering of 1.5 million shares of its common stock. The gross proceeds to 澳门正规博彩十大排行平台 from this offering were expected to be approximately $119.3 million before deducting underwriting discounts and commissions and estimated offering expenses payable by 澳门正规博彩十大排行平台.
澳门正规博彩十大排行平台 Announces 澳门十大博彩正规游戏平台 Approval for Vimizim(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome
The U.S. Food and Drug Administration in February approved Vimizim™ (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.
澳门正规博彩十大排行平台 Announces Agreement With Repligen for Pre-clinical Compounds
澳门正规博彩十大排行平台 announced in January that it has entered into an agreement to purchase Repligen Corporation’s histone deacetylase inhibitor (HDACi) library and related intellectual property. Potential applications of the HDACi portfolio include Friedreich’s ataxia and other neurological disorders.
澳门正规博彩十大排行平台 Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia
澳门正规博彩十大排行平台 in January announced today that it had dosed the first child in the Phase 2 trial with BMN 111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of children with achondroplasia. Achondroplasia is the most common form of disproportionate short stature or dwarfism.
澳门正规博彩十大排行平台 Announces Selection of Factor VIII Gene Therapy Drug Development Candidate BMN 270 for the Treatment of Hemophilia A
澳门正规博彩十大排行平台 announced in January that it has selected an AAV-factor VIII vector, BMN 270, to develop for the treatment of hemophilia A and has initiated IND-enabling studies.
2013
澳门十大博彩正规游戏平台 Advisory Committee Recommends Approval for 澳门正规博彩十大排行平台’s Vimizim™ for the Treatment of Patients with Morquio A Syndrome
In November, 澳门正规博彩十大排行平台 announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) voted in favor of approval of Vimizim for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).
澳门正规博彩十大排行平台 Announces French ATU Granted for Vimizim™ for the Treatment of Morquio A Syndrome
In November, 澳门正规博彩十大排行平台 announced that the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d’Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use, for patient sales of Vimizim for the treatment of Morquio A Syndrome. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists.
澳门正规博彩十大排行平台 Doses First Patient in Phase 1/2 Trial with BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease
In September, 澳门正规博彩十大排行平台 announced that it dosed the first patient in the Phase 1/2 trial for BMN 190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. This is the first time that a patient with Batten disease has been treated with an enzyme replacement therapy in a clinical trial setting.
澳门正规博彩十大排行平台 Appoints Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs
In September, 澳门正规博彩十大排行平台 announced the appointment of Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs.
澳门正规博彩十大排行平台 Initiates Phase 3 Trial for PEG-PAL for the Treatment of PKU
In June, 澳门正规博彩十大排行平台 announced that it initiated the Phase 3 program for PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU).
澳门十大博彩正规游戏平台 Accepts Vimizim BLA and Grants Priority Review Designation
In May, 澳门正规博彩十大排行平台 announced that the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) accepted for review the Biologics License Application (BLA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.
Vimizim MAA Validated by the EMA
In May, 澳门正规博彩十大排行平台 announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Vimizim. Validation of the MAA confirms that the submission is complete and starts the EMA’s formal review process.
澳门正规博彩十大排行平台 Advances BMN-701 for Pompe Disease to Next Phase of Development
In March, 澳门正规博彩十大排行平台 announced results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease.
澳门正规博彩十大排行平台 Licenses Factor VIII Gene Therapy Program for Hemophilia A From University College London and St. Jude Children’s Research Hospital
In February, 澳门正规博彩十大排行平台 announced that it had licensed a Factor VIII gene therapy program for hemophilia A from University College London (UCL) and St. Jude Children’s Research Hospital.
澳门正规博彩十大排行平台 Announces Kuvan Significantly Improves Inattentiveness in Kuvan Responding PKU Patients
In February, 澳门正规博彩十大排行平台 announced results from the PKU-016 ASCEND study, the largest randomized controlled trial evaluating neuropsychiatric outcomes in phenylketonuria (PKU) patients treated with the approved drug Kuvan (sapropterin dihydrochloride).
澳门正规博彩十大排行平台 Acquires Zacharon Pharmaceuticals
In January, 澳门正规博彩十大排行平台 announced that it acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego, focused on developing small molecules targeting pathways of glycan and glycolipid metabolism.
2012
澳门正规博彩十大排行平台 Phase 3 Study of GALNS for the Treatment of MPS IVA Meets Primary Endpoint
In November, 澳门正规博彩十大排行平台 announced that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg.
澳门正规博彩十大排行平台 Announces Decision to Start Phase 3 Program for PEG-PAL
In September, 澳门正规博彩十大排行平台 announced preliminary results from the Phase 2 program of PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU) demonstrating long-term retention, tolerability and providing evidence of efficacy. Based on these results, the company expects to start a pivotal Phase 3 study in the second quarter of 2013, following an anticipated end of Phase 2 meeting with the 澳门十大博彩正规游戏平台 in the first quarter of 2013.
澳门正规博彩十大排行平台 Announces Phase 1 Results for BMN-111 for Achondroplasia
In September, 澳门正规博彩十大排行平台 announced the completion of a Phase 1 study for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia.
澳门正规博彩十大排行平台 Names Jeff Ajer Senior Vice President, Chief Commercial Officer
In September, 澳门正规博彩十大排行平台 announced that Jeff Ajer, the previous Vice President, Commercial Operations, the Americas, was promoted to Senior Vice President, Chief Commercial Officer.
澳门正规博彩十大排行平台 Appoints Dan Spiegelman as Executive Vice President and Chief Financial Officer
In May, 澳门正规博彩十大排行平台 announced the appointment of Dan Spiegelman as Executive Vice President and Chief Financial Officer (CFO).
澳门正规博彩十大排行平台 Completes Enrollment for Phase 3 Trial for GALNS for the Treatment of MPS IVA
In March, 澳门正规博彩十大排行平台 announced that enrollment was complete for the pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN-110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.
澳门正规博彩十大排行平台 Initiates Phase 1 Trial for BMN-111 for the Treatment of Achondroplasia
In February, 澳门正规博彩十大排行平台 announced the initiation of a Phase 1 study in healthy volunteers for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of achondroplasia.
2011
澳门正规博彩十大排行平台 Receives Positive Opinion from European Regulatory Authorities for Expanded Biologics Manufacturing Facility
In December, 澳门正规博彩十大排行平台 announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for the company’s manufacturing facility expansion in Novato, CA.
澳门正规博彩十大排行平台 Announces BMN-190 for Late infantile neuronal ceroid lipofuscinosis (LINCL) – Form of Batten Disease
In December, at its R&D Day, 澳门正规博彩十大排行平台 announced a new clinical program, BMN-190 for LINCL, one form of Batten disease.
澳门正规博彩十大排行平台 Announces Buy Back of Naglazyme Royalties From Adelaide Health Authority
In November, 澳门正规博彩十大排行平台 completed the buy back of certain intellectual property from SA Pathology, a unit of the Central Adelaide Local Health Network located in Adelaide, Australia for an upfront payment of $81 million. The intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between 2022 and 2023.
澳门正规博彩十大排行平台 Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA
In November, 澳门正规博彩十大排行平台 initiated a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA).
澳门正规博彩十大排行平台 Announces 澳门十大博彩正规游戏平台 Approval of Expanded Biologics Manufacturing Facility
In November, 澳门正规博彩十大排行平台 announced tthat it had received approval from the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for its manufacturing facility expansion in Novato, CA.
澳门正规博彩十大排行平台 Agrees to Acquire Biologics Manufacturing Plant in Ireland from Pfizer
In July, 澳门正规博彩十大排行平台 announced that it had entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland. The transaction was completed in August of 2011.
澳门正规博彩十大排行平台 Initiates Phase 3 Trial for Amifampridine Phosphate for the Treatment of LEMS
In June, 澳门正规博彩十大排行平台 initiated a Phase 3 trial for amifampridine phosphate (3,4-diaminopyridine phosphate) for the treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
澳门正规博彩十大排行平台 Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA
In February, 澳门正规博彩十大排行平台 initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.
澳门正规博彩十大排行平台 Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease
In January, 澳门正规博彩十大排行平台 initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.
2010
澳门正规博彩十大排行平台 Announces Program for BMN-111 for the Treatment of Achondroplasia
In October 2010, 澳门正规博彩十大排行平台 announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. 澳门正规博彩十大排行平台 plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012.
澳门正规博彩十大排行平台 Appoints William Young and Kenneth Bate to Its Board of Directors
In September 2010, 澳门正规博彩十大排行平台 announced the appointment of William D. Young and Kenneth M. Bate to its Board of Directors.
澳门正规博彩十大排行平台 Receives Orphan Drug Designation from the 澳门十大博彩正规游戏平台 for BMN-701 for the
Treatment of Pompe Disease
In August 2010, 澳门正规博彩十大排行平台 received orphan drug designation from the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatmen of Pompe disease.
澳门正规博彩十大排行平台 Acquires ZyStor Therapeutics, Inc.
In August 2010, 澳门正规博彩十大排行平台 acquired ZyStor Therapeutics, Inc., a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders.
澳门正规博彩十大排行平台 Initiates Phase 3b Study to Evaluate the Effects of Kuvan on Neurophychiatric
Symptoms in Subjects with PKU
In August 2010, 澳门正规博彩十大排行平台 announced that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU).
澳门正规博彩十大排行平台 Halts Development of BMN-195 for Duchenne Muscular Dystrophy
In August 2010, 澳门正规博彩十大排行平台 completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial results were not positive and the BMN 195 program was discontinued due to pharmaceutical and pharmacokinetic challenges.
澳门正规博彩十大排行平台 Reports Positive Results for Phase I/II Trial for BMN-110 for MPS
IVA
In April 2010, 澳门正规博彩十大排行平台 announced positive results for the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome.
澳门正规博彩十大排行平台 Launches Firdapse in the European Union
In April 2010 澳门正规博彩十大排行平台 launched Firdapse(TM) (3,4-diaminopyridine) in
the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).
澳门正规博彩十大排行平台 Acquires LEAD Therapeutics
In February 2010, 澳门正规博彩十大排行平台 entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers.
澳门正规博彩十大排行平台 Initiates Phase 1 Clinical Study of BMN-195 for Duchenne Muscular
Dystrophy
In January 2010, 澳门正规博彩十大排行平台 announced that the first subject had initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD).
Firdapse Receives Marketing Approval in the EU for LEMS
In January 2010, 澳门正规博彩十大排行平台 announced today that the European Commission had granted marketing approval for Firdapse (3,4-diaminopyridine (amifampridine phosphate), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).
2009
澳门十大博彩正规游戏平台 Grants Orphan Drug Designation for 3, 4-DAP for LEMS
In November 2009, the Food and Drug Administration (澳门十大博彩正规游戏平台) granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS in Europe.
澳门正规博彩十大排行平台 Acquires Huxley Pharmaceuticals, Inc.
In October 2009, the company acquired Huxley Pharmaceuticals, Inc., which has rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). The company expects to launch the product in Europe in the first quarter of 2010, and is evaluating a development strategy for amifampridine phosphate in LEMS in the U.S. and for other indications in the U.S. and Europe.
澳门正规博彩十大排行平台 Initiates Phase II Clinical Study of PEG-PAL in PKU
In September 2009, the company initiated the Phase II clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010. The primary objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU. The secondary objectives are to evaluate the safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels of PEG-PAL.
Kuvan Receives Priority Review Status From Health Canada
In June 2009, Kuvan® (sapropterin dihydrochloride) received priority review status from Health Canada. Priority review provides for a shortened submission review of 180 days versus the standard twelve months. 澳门正规博彩十大排行平台 plans to file a marketing application for Kuvan in Canada in the third quarter of 2009, and with priority review status, a decision for marketing approval is expected in the first half of 2010.
澳门正规博彩十大排行平台 Initiates Phase I/II Clinical Trial for GALNS for Morquio A Syndrome (MPS IVA)
In April 2009, 澳门正规博彩十大排行平台 initiated a Phase 1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies.
澳门正规博彩十大排行平台 Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan
In March 2009, 澳门正规博彩十大排行平台 received a notice from the U.S. Patent Office reporting allowance of claims covering once daily dosing methods for
Kuvan® (sapropterin dihydrochloride) Tablets
in the treatment of phenylketonuria (PKU). The company expects that the patent will be issued later in 2009. If issued, the patents 20-year term would expire in 2024. The company has a number of other pending patent applications covering various aspects of Kuvan compositions and dosing.
澳门正规博彩十大排行平台s Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA
In March 2009, 澳门正规博彩十大排行平台 received formal acceptance from the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for its application for the clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS Type IVA) or Morquio A Syndrome. A Phase 1/2 clinical trial is expected to begin soon.
澳门正规博彩十大排行平台 Appoints Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer
In March 2009, 澳门正规博彩十大排行平台 welcomed Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer (CMO). Dr. Fuchs replaced longtime 澳门正规博彩十大排行平台 CMO, Dr. Emil Kakkis.
Naglazyme Approved by Brazils National Health Surveillance Agency
In March 2009, 澳门正规博彩十大排行平台 received marketing approval from ANVISA, Brazils National Health Surveillance Agency, for Naglazyme® (galsulfase) for the treatment of patients with Mucopolysaccharidosis VI (MPS VI). Estimates indicate that Brazil has the largest known number of affected MPS VI patients in the world.
Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically Significant
In February 2009, 澳门正规博彩十大排行平台 announced results from its Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). There was no statistical significance observed between the 6R-BH4 treatment and placebo groups. 澳门正规博彩十大排行平台 will determine the future of its 6R-BH4 cardiovascular program following the results of additional investigator-sponsored studies of 6R-BH4 including proteinuria, pulmonary arterial hypertension and 6R-BH4 plus vitamin C in patients with endothelial dysfunction.
Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile
In February 2009, 澳门正规博彩十大排行平台 and La Jolla Pharmaceutical (LJP) announced that in the first interim efficacy analysis (IEA) for the Riquent® Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial was futile. Following this analysis, 澳门正规博彩十大排行平台 and La Jolla discontinued the study and 澳门正规博彩十大排行平台 opted-out of its agreement with La Jolla to develop and commercialize Riquent in the United States, Europe and all other territories of the world, excluding the Asia Pacific region.
澳门正规博彩十大排行平台 & La Jolla Pharmaceutical Sign Worldwide Development and Commercialization Agreement for Riquent
In January 2009, 澳门正规博彩十大排行平台 and La Jolla Pharmaceutical entered into an agreement to develop and commercialize Riquent®, La Jolla’s investigational drug for lupus nephritus in the United States, Europe and all other territories of the world, excluding the Asia Pacific region. If the Phase 3 trial is successful and 澳门正规博彩十大排行平台 opts-in the parties will share equally in all losses and profits. In the United States, 澳门正规博彩十大排行平台 and La Jolla will share Riquent commercialization rights. In Europe and other territories outside of Asia, 澳门正规博彩十大排行平台 will have exclusive rights to commercialize the product.
2008
Kuvan Approved By EMEA for Use in Europe
In December, the scientific committee of the European Medicines Agency (EMEA) approved Kuvan® (sapropterin dihydrochloride) Tablets as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. This approval triggered a $30 million milestone payment to 澳门正规博彩十大排行平台 from partner Merck Serono.
澳门正规博彩十大排行平台 Initiates Clinical Program for Morquio A Syndrome (MPS IVA)
In November, 澳门正规博彩十大排行平台 initiated a clinical assessment program (MorCAP) for patients with MPS IVA (Morquio A Syndrome). Data collected will be used to augment current understanding of the disease by measuring endurance, respiratory function and other parameters in affected patients.
Positive Results from Study of 6R-BH4 in Sickle Cell Disease
In October, positive results from a Phase 2a multi-center, open-label, dose-escalation clinical study of 6R-BH4 in patients with sickle cell disease (SCD) indicate that oral administration of 6R-BH4 is associated with improvements in endothelial dysfunction in sickle cell disease patients. Endothelial dysfunction was measured using the EndoPAT device to assess peripheral arterial tonometry (PAT), which is commonly used in assessing the sickle cell disease patient population.
Kuvan Receives Positive Opinion from CHMP for European Approval
In September, 澳门正规博彩十大排行平台’s partner Merck Serono received a positive opinion for Kuvan® (sapropterin dihydrochloride) Tablets as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMEA). The CHMP recommendation will be considered by the European Commission, which will deliver its final decision on the granting of marketing authorization within 67 days. A positive opinion will trigger a $30 million milestone payment to 澳门正规博彩十大排行平台.
澳门正规博彩十大排行平台 and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program
In July, 澳门正规博彩十大排行平台 and Summit Corporation partnered in an exclusive worldwide licensing agreement for Summit’s novel preclinical candidate SMT C1100 and all follow-on molecules, which are being developed to treat the fatal genetic disorder Duchenne Muscular Dystrophy (DMD).
Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU
In July, 澳门正规博彩十大排行平台 partner, Asubio Pharma Co., Ltd., received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label extension of Biopten® (sapropterin dihydrochloride), which contains the same active ingredient as Kuvan® for the treatment of patients with phenylketonuria (PKU).
澳门正规博彩十大排行平台 Announces Program for ERT for Treatment of MPS IVA – Morquio A Syndrome
In June, 澳门正规博彩十大排行平台 announced a new program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome.
澳门正规博彩十大排行平台 Initiates Phase 1 Clinical Study of PEG-PAL in PKU
In May, the first patient initiated treatment in 澳门正规博彩十大排行平台’s Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU).
Naglazyme Approved by Japanese Ministry of Health
In March, AnGes MG, Inc., 澳门正规博彩十大排行平台’s marketing and distribution partner in Japan, received approval for its Marketing Application for Naglazyme® (galsulfase) from the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with mucopolysaccharidosis VI ( MPS VI).
澳门正规博彩十大排行平台 and Genzyme Restructure Aldurazyme 50/50 Joint Venture
In January, 澳门正规博彩十大排行平台 and Genzyme announced a restructuring of their joint venture regarding Aldurazyme® (laronidase). Genzyme will continue to globally market and sell Aldurazyme for mucopolysaccharidosis I (MPS I) and 澳门正规博彩十大排行平台 will continue to manufacture Aldurazyme. Under the revised structure, payments are projected to result in both 澳门正规博彩十大排行平台 and Genzyme receiving approximately the same profit as under the original joint venture structure.
2007
澳门正规博彩十大排行平台 Re-Acquires Rights To Kuvan In Canada From Merck Serono
In December, 澳门正规博彩十大排行平台 re-acquired rights to Kuvan in Canada, which will enable the company to better coordinate commercialization efforts in the North American market. Terms of the agreement specify a reduction in royalties owed to 澳门正规博彩十大排行平台 on Merck Serono sales outside the United States and Japan
Kuvan Approved By 澳门十大博彩正规游戏平台; Launched Immediately In The U.S.
In December, approximately three years after filing the IND, 澳门正规博彩十大排行平台 received 澳门十大博彩正规游戏平台 approval for Kuvan® (sapropterin dihydrochloride) Tablets, the first specific drug therapy for the treatment of phenylketonuria (PKU). The product was launched in the U.S. immediately following the 澳门十大博彩正规游戏平台 approval. Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4) responsive PKU and is to be used in conjunction with a Phe-restricted diet.
澳门正规博彩十大排行平台 And IGAN Collaborate On Development Of Enzyme Therapy To Treat IgA Nephropathy
In December, 澳门正规博彩十大排行平台 and IGAN Biosciences initiated a program to develop an IgA protease for treating IgA nephropathy, an orphan designated kidney disorder with few treatment alternatives. In the United States, approximately 800 patients per year develop end stage renal disease caused by IgA nephropathy. An estimated 40,000 are affected by the disorder.
澳门正规博彩十大排行平台 Files IND For PEG-PAL For The Treatment Of PKU
In November, 澳门正规博彩十大排行平台 filed an investigational new drug application (IND) with the 澳门十大博彩正规游戏平台 for PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), formerly known as Phenylase™, for the treatment of phenylketonuria (PKU). The company expects to initiate a clinical study of PEG-PAL in PKU patients in the first quarter of 2008. Preclinical data has demonstrated that PEG-PAL administered subcutaneously once weekly to PKU mice resulted in a sustained decrease in blood phenylalanine (Phe) levels in a twelve week study and has also shown potent Phe level reductions in primates.
MAA For Sapropterin For Hyperphenylalaninemia Submitted To EMEA
In November, 澳门正规博彩十大排行平台 partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride (known as Kuvan® in the U.S.) an oral treatment for patients suffering from significant hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. Acceptance of the MAA filing by the EMEA will trigger a $15 million milestone payment to 澳门正规博彩十大排行平台. Sapropterin has received orphan medicinal product designation in the European Union.
Exclusive Rights To Kuvan Data Licensed To Asubio Pharma
In September, 澳门正规博彩十大排行平台 has licensed exclusive rights to data and intellectual property contained in the Kuvan (sapropterin dihydrochloride) Tablets NDA to long-standing partner, Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo). Asubio will use this data to supplement its current filing to the Japanese Ministry of Health, Labour and Welfare for approval of its BH4 product for the treatment of PKU in Japan. This new data greatly expands the clinical data set on treatment of PKU and is expected to accelerate the timing for the label extension of Asubio’s current BH4 product. 澳门正规博彩十大排行平台 will receive a milestone payment for approval as well as double-digit royalties on net sales of BH4 for PKU in Japan.
BLA For Naglazyme Submitted To Japanese Ministry Of Health
In August, AnGes MG, Inc. (AnGes), 澳门正规博彩十大排行平台’s marketing and distribution partner in Japan submitted a Biologics License Application (BLA) for Naglazyme® (galsulfase) to the Japanese Ministry of Health, Labour and Welfare. If approved, Naglazyme will be the first drug treatment option to MPS VI patients in Japan. Naglazyme has obtained an orphan designation in Japan.
Kuvan Receives Priority Review Status From 澳门十大博彩正规游戏平台
In July, priority review status is an 澳门十大博彩正规游戏平台 designation granted to drugs that, if approved, will provide a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease.
澳门正规博彩十大排行平台 Initiates Expanded Access Program For Kuvan In The U.S.
In June, the first patient initiated treatment in 澳门正规博彩十大排行平台’s expanded access program for Kuvan. Under an expanded access program, the 澳门十大博彩正规游戏平台 allows early access to investigational drugs being developed to treat serious diseases for which there is no satisfactory alternative therapy. 澳门正规博彩十大排行平台 will provide Kuvan at no charge to up to 500 U.S. patients throughout the duration of the program.
澳门正规博彩十大排行平台 Submits NDA For Kuvan For PKU
In May, the NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents 澳门正规博彩十大排行平台’s largest and most comprehensive filing to date. The fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. If the 澳门十大博彩正规游戏平台 accepts the NDA and grants the request for priority review, the 澳门十大博彩正规游戏平台 is expected to take action on the application within six months of its submission. Kuvan has received the orphan drug designation, which allows for seven years of market exclusivity within the United States following 澳门十大博彩正规游戏平台 approval.
澳门正规博彩十大排行平台 Initiates Phase 2a Clinical Study Of 6R-BH4 In Sickle Cell Disease
In May, the first patient initiated treatment in the Phase 2a clinical study of 6R-BH4 (sapropterin dihydrochloride) for the treatment of sickle cell disease (SCD). SCD is an orphan disease with 70,000 to 100,000 patients in the U.S. It is well-diagnosed at birth, but there is only one approved drug treatment option currently available which is used by a minority of patients due to toxicity problems. The Phase 2a multi-center, open-label study will evaluate the safety of oral 6R-BH4 administered in escalating doses in patients with sickle cell disease, as well as changes in physiological and biochemical markers of endothelial function which underlie some key aspects of SCD.
Results From Phase 2 Clinical Study Of 6R-BH4 In Poorly Controlled Hypertension
In February, results demonstrated that there was no statistically significant or clinically meaningful effect of 6R-BH4 on any efficacy or safety parameter measured, relative to placebo, despite encouraging pre-clinical and clinical studies of 6R-BH4 in diseases with endothelial dysfunction.
Positive Results From Phase 3 Diet Study Of Phenoptin For PKU
In January, all pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled Phase 3 diet study of Phenoptin™ (sapropterin dihydrochloride) were met. Treatment resulted in a significant increase in patients’ phenylalanine tolerance as well as a reduction in their blood phenylalanine levels. In addition, the data showed that Phenoptin was well tolerated in younger PKU patients under dietary control.
Phase 2 Clinical Study Of 6R-BH4 In Peripheral Arterial Disease Initiated
In January, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of symptomatic peripheral arterial disease. The company expects to announce data from this study in the first half of fiscal year 2008. Peripheral arterial disease results from endothelial dysfunction and affects approximately eight million Americans, many of whom also suffer from intermittent claudication. The Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study is designed to compare oral 6R-BH4 to placebo in subjects with intermittent claudication (IC) caused by peripheral arterial disease.
2006
Positive Results From Phase 3 Extension Study Of Phenoptin For PKU
In December, data has confirmed the long-term safety, tolerability, and efficacy of Phenoptin to control blood Phe levels across a range of doses in PKU patients. The company is on track to file the NDA in the second quarter of 2007. A once daily dose regimen of Phenoptin was sufficient to maintain the reduction of blood Phe levels throughout a 24-hour period. The incidence and type of adverse events were comparable to that of the placebo group during the double-blind study and nearly all were mild or moderate in severity.
Aldurazyme Receives Marketing Approval In Japan
In October, Japan’s Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Aldurazyme® (laronidase), the first specific treatment for MPS I approved in Japan. Aldurazyme has been designated as an orphan drug in Japan.
Orapred ODT Launched By Alliant Pharmaceuticals
In August, Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), the first 澳门十大博彩正规游戏平台-approved orally disintegrating tablet form of prednisolone, is now available in the United States. The Orapred product line, which includes Orapred ODT and Orapred® (prednisolone sodium phosphate oral solution) is marketed by Alliant Pharmaceuticals, Inc. Under the terms of the marketing agreement, 澳门正规博彩十大排行平台 will receive milestone payments and royalties on Orapred products sales. 澳门正规博彩十大排行平台 will retain commercial rights to the Orapred product line outside of North America.
Phase 2 Clinical Study Of 6R-BH4 In Poorly Controlled Hypertension Initiated
In July, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of poorly controlled hypertension. The company hopes to announce data from this study in early 2007 that will confirm results seen earlier in pilot clinical studies that demonstrated that oral administration of 6R-BH4 can reduce blood pressure in individuals who remain hypertensive despite treatment with other medications.
澳门正规博彩十大排行平台 And Alliant Pharmaceuticals Establish North American Licensing Agreement For Orapred
In March, the licensing and acquisition agreement provides exclusive North American rights to Alliant for the Orapred® (prednisolone sodium phosphate oral solution) product line, including Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets). 澳门正规博彩十大排行平台 will receive payments and royalties based on the product’s approval, launch and level of sales. 澳门正规博彩十大排行平台 will retain commercial rights outside of North America
Positive Results From Phase 3 Clinical Study Of Phenoptin For PKU
In March, positive results of a Phase 3, double-blind, placebo-controlled clinical study of Phenoptin™ (sapropterin dihydrochloride) confirmed that all pre-specified primary and secondary endpoints were met. Data demonstrates a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo.
Naglazyme Receives European Union Approval
In January, the European Commission has granted marketing authorization for Naglazyme® (galsulfase), the first treatment for MPS VI approved in the European Union. Naglazyme has been granted orphan drug status in the EU, which confers 10 years of market exclusivity. 澳门正规博彩十大排行平台 will launch the product on a country-by-country basis.
Phenoptin For PKU Receives 澳门十大博彩正规游戏平台 Fast Track Designation
In January, the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台) granted Fast Track designation for Phenoptin™ (sapropterin dihydrochloride) for PKU, currently in Phase 3 clinical development.
The Fast Track program is designed to expedite the development and review process of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
澳门正规博彩十大排行平台 Establishes Commercial Operations
In Europe
In January, in anticipation of the pending European marketing approval for Naglazyme™, commercial operations have been established in Europe. 澳门正规博彩十大排行平台 Europe Ltd., headquartered in London, with branch offices located in Spain, Switzerland and Italy, will be responsible for overseeing the sales and distribution of Naglazyme to the 25 member states of the European Union, Iceland and Norway.
2005
澳门正规博彩十大排行平台 Files New Drug Application For
Orapred ODT
In August, 澳门正规博彩十大排行平台 submitted a New Drug Application to the 澳门十大博彩正规游戏平台 for Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), a new formulation of Orapred® (prednisolone sodium phosphate oral solution). Prednisolone is commonly used to reduce inflammation seen in numerous medical conditions including asthma, arthritis, and cancer.
Orapred ODT may have the potential to provide individuals of all ages with a new formulation of prednisolone that is convenient and easy to administer.
澳门正规博彩十大排行平台 Launches Naglazyme In The United
States
In June, Naglazyme® is the first drug therapy independently developed and commercialized by 澳门正规博彩十大排行平台. A team of U.S. based medical science liaisons will be responsible for providing support to infusion centers and physicians who administer Naglazyme.
澳门正规博彩十大排行平台 Receives 澳门十大博彩正规游戏平台 Approval For Naglazyme
In May, Naglazyme®, represents 澳门正规博彩十大排行平台’s first independently developed and commercialized drug and the first 澳门十大博彩正规游戏平台-approved treatment for MPS VI. The drug was granted the orphan drug designation in the United States, which confers seven years of market exclusivity.
澳门正规博彩十大排行平台 Forms Strategic Alliance With Serono for the Development and Commercialization of Phenoptin and Phenylase
In May, 澳门正规博彩十大排行平台 and Serono finalize an egreement to help fund late-stage development of 澳门正规博彩十大排行平台’s PKU and 6R-BH4 programs. The companies will equally share Phase 3 development costs of Phenoptin and Phenylase for PKU and 6R-BH4 for the treatment of cardiovascular indications. Additionally, Serono will provide 澳门正规博彩十大排行平台 up to $232 million in milestone payments in exchange for ex-U.S. commercialization rights (excluding Japan).
澳门正规博彩十大排行平台 Acquires Rights to 6R-BH4 for the Treatment of Cardiovascular Indications
In May, 澳门正规博彩十大排行平台 announced a partnership with Daiichi Suntory Pharma Co., Ltd. that gave the company exclusive worldwide rights (excluding Japan) for the use of 6R-BH4 to treat cardiovascular indications. This was the second agreement reached with Daiichi; the first, which was reached in November 2004, pertained to intellectual property, preclinical and clinical data on 6R-BH4 for genetic disorders including PKU, and to manufacturing and supply of 6R-BH4.
澳门正规博彩十大排行平台 Initiates Phase 3 Clinical Study of Phenoptin for PKU
In April, 澳门正规博彩十大排行平台 initiated a Phase 3 clinical study designed to evaluate the safety and efficacy of Phenoptin for the treatment of PKU. Phenoptin has been designated an orphan drug in the United States and Europe and assigned Fast Track status in the United States.
2004
澳门正规博彩十大排行平台 Files Marketing Applications for Naglazyme in the United States and Europe
In June, 澳门正规博彩十大排行平台 announced positive results of the Phase 3 clinical trial of Naglayzme (galsulfase) for MPS VI, keeping the company on track to file license applications in both the United States and European Union by the close of the year.
澳门正规博彩十大排行平台 Advances Phenoptin for PKU into Phase 2 Development
In February, 澳门正规博彩十大排行平台 advanced Phenoptin™ (sapropterin dihydrochloride), an investigational small-molecule oral therapeutic for the treatment of phenylketonuria (PKU), from IND filing into Phase 2 development by the close of the year.
2003
Aldurazyme for MPS I Approved and Launched in the United States and Europe
In April and June, respectively, the 澳门十大博彩正规游戏平台 and European Commission (EC) granted marketing authorization for Aldurazyme–the first approved enzyme replacement therapy for the treatment of MPS I. Aldurazyme received 澳门十大博彩正规游戏平台 approval in just over five and a half years after the investigational new drug application (IND) was filed.
2000
澳门正规博彩十大排行平台 Initiates Clinical Trial of Naglazyme for MPS VI
At the onset of clinical development, Naglazyme® (galsulfase) for MPS VI had been granted orphan drug and fast track designations by the U.S. Food and Drug Administration (澳门十大博彩正规游戏平台). The company manufactured the investigational enzyme at its cGMP manufacturing facility located near the large-scale facility where it is being manufactured today.
1999
澳门正规博彩十大排行平台 Becomes a Publicly Traded Company (Nasdaq/SWX:BMRN)
In July, 澳门正规博彩十大排行平台 completed an initial public offering, raising $67.3 million. Since a large number of the early investors were based in Europe, the company was listed on the Swiss SWX Exchange in addition to the Nasdaq National Market.
1998
澳门正规博彩十大排行平台/Genzyme LLC Formed to Support the Development and Commercialization of Aldurazyme
In September, 澳门正规博彩十大排行平台 and Genzyme Corporation established 澳门正规博彩十大排行平台/Genzyme LLC, a 50/50 joint venture for the worldwide development and commercialization of Aldurazyme for MPS I. Pursuant to the agreement, 澳门正规博彩十大排行平台 is responsible for manufacturing the product and Genzyme is responsible for its commercialization. All expenses and profits and are shared equally between the companies.
1997
澳门正规博彩十大排行平台 Initiates Clinical Trial of Aldurazyme for MPS I
In December, 澳门正规博彩十大排行平台 initiated the first clinical trial of Aldurazyme® (laronidase) for the treatment of mucopolysaccharidosis I (MPS I). Dr. Emil Kakkis, then a fellow at Harbor-UCLA Medical Center, was the studys principal investigator and Dr. Elizabeth Neufeld, Professor and Chair of the Biological Chemistry Department at UCLA, was the advisor. Together, Dr. Neufeld and Dr. Kakkis discovered how to produce a recombinant form of alpha-L-iduronidase that was later evaluated in the clinic.
澳门正规博彩十大排行平台 Pharmaceutical Inc. Founded
In March, with a $1.5 million investment from Glyko Biomedical Ltd., 澳门正规博彩十大排行平台 was open for business. The company’s mission was to leverage its proprietary enzyme technology to develop therapies for the treatment of numerous diseases and conditions including genetic diseases, and burn and wound care. By the close of the year, the company had raised an additional $11.3 million from private investors.