Sarepta Grabs Accelerated Approval for DMD Therapy

Sarepta Therapeutics’ Vyondys 53 (golodirsen) injection earned accelerated approval from the FDA to treat certain Duchenne muscular dystrophy (DMD) patients.

The approval was based on a clinical study in which Vynodys 53 demonstrated increased dystrophin levels after 48 weeks of treatment.

The drug received many designations, including fast track, priority review, orphan drug, and rare pediatric disease priority review.

As a condition of its accelerated approval, the agency is requiring Sarepta to conduct a clinical trial to assess if the drug improves motor function of DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 53 skipping.