Tag Archives: BioMarin

May, 2018

25 May
FDA Approves Biomarin’s Palynziq Injection for Treatment of Phenylketonuria, A Rare Genetic Disease

SAN RAFAEL, Calif., May 24, 2018 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that BioMarin received standard approval from the U.S. Food and Drug Administration (FDA) 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 …
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27 November
Biomarin Sells Rare Pediatric Disease Priority Review Voucher for $125 Million

SAN RAFAEL, Calif., Nov. 27, 2017 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into a definitive agreement to sell the Rare Pediatric Disease Priority Review Voucher (PRV) it obtained in April of this year for a lump sum payment of $125,000,000. The Company received …
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27 October
Biomarin’s Gene Therapy Granted FDA Breakthrough Therapy Designation for the Treatment of Hemophilia A

SAN RAFAEL, Calif., Oct. 26, 2017 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation. The FDA’s Breakthrough Therapy Designation program is intended to facilitate and expedite development and review of new drugs …
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27 April
FDA Approves Biomarin’s Brineura as the First Treatment for a Form of Batten Disease

SAN RAFAEL, Calif., April 27, 2017 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) 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), …
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21 March
BioMarin’s PKU Drug Demonstrates Mixed Results in Late-Stage Study

SAN RAFAEL, Calif., March 21, 2016 (GLOBE NEWSWIRE) — BioMarin announced today 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 …
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14 January
FDA Rejects Biomarin’s Duchenne Muscular Dystrophy Drug

SAN RAFAEL, Calif., Jan. 14, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) 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 …
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25 November
FDA Panel Says BioMarin DMD Drug Data Not Persuasive

SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for …
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