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BioMarin Announces FDA Advisory Committee to Review Drisapersen for Treatment of Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Advisory Committee Meeting Scheduled for November 24, 2015

SAN RAFAEL, Calif., Oct. 15, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for drisapersen. The FDA is currently reviewing drisapersen for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping. The advisory committee will review drisapersen data included in a new drug application (NDA) during a meeting on November 24, 2015.

"Duchenne muscular dystrophy is a deadly, progressive muscle disorder with limited treatment options that affects mostly boys and young men. We welcome the necessary regulatory review processes to move treatment for this community beyond supportive care to a therapy that addresses the underlying cause of the disease," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance. "We look forward to discussing the New Drug Application for drisapersen with the advisory committee with the goal of bringing this much-needed treatment one step closer to patients who have a specific type of Duchenne muscular dystrophy."

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy patients amenable to single exon skipping. In the U.S., it is estimated there are approximately 2,000 patients who might benefit from treatment with drisapersen.

The Prescription Drug User Fee Act (PDUFA)...


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