GlaxoSmithKline received verbal notification that the FDA has granted Breakthrough Therapy designation to its investigational compound drisapersen (previously GSK2402968/PRO051) for the potential treatment of patients with Duchenne Muscular Dystrophy, a debilitating neuromuscular disease. Drisapersen is an antisense oligonucleotide which induces skipping exon-51.

The Breakthrough Therapy designation was based on results from a Phase 2 study (DMD114117). GSK’s clinical development plan evaluates the effect of drisapersen in ambulant (Phases 2 and 3) and non-ambulant boys (Phase 1) with Duchenne Muscular Dystrophy who have dystrophin gene mutations amenable to an exon 51 skip. Up to 13% of boys with Duchenne Muscular Dystrophy have dystrophin gene mutations/deletions amenable to an exon 51 skip.

Duchenne Muscular Dystrophy, a progressive muscle wasting disease, is the most common and lethal form of muscular dystrophy for which there is no treatment. Duchenne impacts one in every 3,500 boys; nearly 20,000 boys are living with the disease in the United States. Boys with Duchenne are usually diagnosed before the age of 5 and are in a wheelchair by age 12 while most do not survive their mid-20s.

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