BioMarin ( BioMarin )


BioMarin's picture

The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; KUVAN® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS).

BioMarin press release, blog etc

07/09/2018 - 09:22 BioMarin Partners with Believe Limited for 'Breaking Through!' Musical Theater Intensive
06/27/2018 - 19:44 BioMarin Announces First Recipients of RARE Scholars Scholarship Program
05/24/2018 - 16:44 BioMarin Receives Standard Approval for Palynziq (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease
05/17/2018 - 16:01 BioMarin Announces 1 Platform and 3 Poster Presentations at World Federation of Hemophilia 2018 World Congress
05/15/2018 - 07:38 BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies
05/01/2018 - 13:39 BioMarin Named To Forbes List Of America's Best Mid-size Employers
04/24/2018 - 12:08 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
03/28/2018 - 11:07 European Medicines Agency (EMA) Accepts BioMarin's Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU)
03/28/2018 - 00:37 (pegvaliase-pqpz) Injection for PKU
03/21/2018 - 04:14 BioMarin's Gene Therapy Manufacturing Facility Recognized with Industry Award
02/28/2018 - 09:35 Rare Disease Day 2018: BioMarin Launches RARE Scholars Scholarship Program
02/07/2018 - 10:59 BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium 2018
02/06/2018 - 06:28 WORLDSymposium Recognizes BioMarin's Brineura (cerliponase alfa) with 2018 New Treatment Award
02/05/2018 - 15:05 BioMarin Announces 20 Presentations at 14th Annual WORLDSymposium 2018
12/22/2017 - 08:49 BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018
12/19/2017 - 19:54 BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
12/11/2017 - 12:13 BioMarin Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting
12/09/2017 - 08:24 BioMarin 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