SAN RAFAEL, Calif., Nov. 20, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the U.S. Food and Drug Administration (FDA) posted briefing materials prepared by BioMarin and by the FDA. The materials pertain to the November 24, 2015 Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) meeting to review the New Drug Application (NDA) for Kyndrisa™ (drisapersen) for the potential treatment of Duchenne muscular dystrophy (Duchenne) amendable to exon 51 skipping.
The PCNS advisory meeting is scheduled for November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials and webcast information can be found on the FDA website at:
http://www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm
The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the Kyndrisa NDA is December 27, 2015.
About Duchenne Muscular Dystrophy
Changes in the dystrophin gene (mutations) that lead to the near absence of dystrophin protein result in the most severe form of dystrophin deficient muscular dystrophy, Duchenne muscular dystrophy, also known as just Duchenne. Boys living with Duchenne experience progressive muscle weakness, causing serious medical complications including serious heart or respiratory-related complications, resulting in death in early adulthood.
Primarily affecting boys, Duchenne affects approximately 1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood.
There is currently no FDA approved therapy designed specifically to treat Duchenne.
About Kyndrisa and Exon Skipping
Kyndrisa is an antisense oligonucleotide that induces exon skipping to provide a molecular patch for dystrophin transcripts produced by certain mutated dystrophin genes. Exons are the parts of a gene that contain the instructions for generating a protein. In applicable cases, skipping an exon near the mutation allows for the production of a truncated but functional dystrophin protein.
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company’s portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.BMRN.com.
