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news.GlaxoSmithKline's investigational compound drisapersen is being granted Breakthrough Therapy status by the US Food and Drug Administration (FDA) for the potential treatment of patients suffering from Duchenne Muscular Dystrophy (DMD).
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The Breakthrough Therapy designation has been granted by the regulator, after vetting the results from the Phase II Study (DMD114117), submitted in April at Cold Spring Harbor.
The phase II study reported that boys taking drisapersen were able to walk 35m more than those on placebo, with the difference maintained up to 48 weeks.
Drisapersen, which will be used in the treatment of DMD caused by mutations in the dystrophin gene, is being developed by GlaxoSmithKline, licensed from Dutch company Prosensa Therapeutics.
With an aim to expedite the development and review of drugs for serious or life-threatening conditions, the FDA created the Breakthrough Therapy designation and enacted it in 2012.
Commenting on the FDA approval, CureDuchenne CEO and Founder Debra Miller said, "The Breakthrough Therapy designation means the FDA has reviewed the data for drisapersen and will provide additional resources."
"These results validate our early efforts to fund novel research and offer hope for finding an effective and safe treatment for all those living with Duchenne," Miller added.
CureDuchenne is engaged in raising awareness and funding research to find a cure for Duchenne disease.