FDA Advisory Committee to review drisapersen for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping- BioMarin

BioMarin Pharmaceutical has announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the 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 24 November 2015.

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 US, it is estimated there are approximately 2,000 patients who might benefit from treatment with drisapersen. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the drisapersen NDA is 27 December 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. The FDA has also granted drisapersen Orphan and Fast Track status, as well as Breakthrough Therapy designation.