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FDA U-turns to approve Sarepta's Vyondys 53 in Duchenne muscular dystrophy

Published on 13/12/19 at 12:00pm

The FDA has surprised industry pundits by choosing to approve Sarepta’s Vyondys 53 under an accelerated pathway for the treatment of Duchenne muscular dystrophy (DMD) just four months after it announced it would be rejecting the drug.

Doubly surprising was the fact that there had been no reports that the company had resubmitted its application for the drug. The application covers the approximately 8% of DMD patients with a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

This decision was based on data showing that Vyondys 53 increased dystrophin production in skeletal muscle in some patients that is “reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping”; the drug increased dystrophin levels to 1.02% of normal from 0.10% at baseline following 48 weeks or longer of treatment.  

However, its ability to improve motor function has not yet been confirmed; the FDA has considered these potential risks in its ruling, and has asked Sarepta to provide further data on the drug’s efficacy.

"The FDA recognises the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies and patients to facilitate the development and approval of treatments for rare diseases,” said Dr Billy Dunn, acting Director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research. “With today's accelerated approval, patients with Duchenne muscular dystrophy — a rare and devastating disease — who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype. Use of the accelerated approval pathway will make Vyondys 53 available to patients based on initial data and we look forward to learning more about the drug's clinical benefit from the ongoing confirmatory clinical trial."

Matt Fellows

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