BioMarin Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease

BioMarin Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease

May 24, 2018 First Enzyme Therapy to Treat PKU Palynziq Significantly and Substantially Reduced Blood Phenylalanine Levels Confirmed by a Phase 3 Placebo-Controlled Randomized Withdrawal Study Palynziq Approval Comes During National PKU Awareness Month Conference Call and Webcast to be

BioMarin 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

BioMarin 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

May 22, 2018 Eliminated Need for Prophylaxis and No Spontaneous Bleeds in Year 2 Quality of Life Scores Continue to Increase in Year 2 Protocol of Global GENEr8-1 (Phase 3) Pivotal Study Amended to Evaluate Superiority Compared to Standard of

BioMarin to Attend Upcoming Investor Conference

– Bank of America Merrill Lynch Health Care Conference on May 15 in Las Vegas SAN RAFAEL, Calif., May 2, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will participate in the Bank

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

Less decline in motor and language function compared to historical controls. SAN RAFAEL, Calif., April 24, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the New England Journal of Medicine (NEJM) published updated results from a multi-center, open-label, dose-escalation and ongoing

Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU)

FDA PDUFA Action Date for Pegvaliase is May 25, 2018 SAN RAFAEL, Calif., March 28, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the European Medicines Agency (EMA) has accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase,

BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium™ 2018

Normalization of biomarker and liver size; stabilization of early cognitive effects suggested in preliminary data SAN RAFAEL, Calif., Feb. 7, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that it presented interim data from a Phase 1/2 trial for BMN 250,

BioMarin Announces 20 Presentations at 14th Annual WORLDSymposium™ 2018

Oral Presentations to Highlight Results from Studies for the Treatment of Sanfilippo Syndrome Type B, Morquio A Syndrome and CLN2 Disease, a Form of Batten Disease… SAN RAFAEL, Calif., Feb. 5, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that