The U.S. Food and Drug Administration granted accelerated approval to Sarepta Therapeutics Inc.'s Vyondys 53 injection to treat Duchenne muscular dystrophy after previously rejecting the drug.
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness.
Vyondys 53 was approved to treat DMD patients with confirmed mutation of the dystrophin gene amenable to exon 53 skipping. According to the regulator, the drug is the first treatment for this specific subtype of the disease, which affects about 8% of DMD patients.
Sarepta is conducting a confirmatory trial, dubbed Essence, to confirm the drug's predicted clinical benefit. The company expects the study, which is enrolling patients, to be completed by 2024.
In August, the FDA issued a complete response letter for Sarepta's new drug application for Vyondys 53, or golodirsen, injection. The regulator was concerned about the risk of infections related to intravenous infusion ports and kidney-related toxicity seen in preclinical models of the injection.
Following the rejection, the Cambridge, Mass.-based company appealed the FDA's decision and resubmitted the NDA.
Sarepta's Exondys 51 also treats DMD but targets a different section of the gene compared to Vyondys 53.
Sarepta's stock was up by 27.9% to $128.5 as of 7:39 p.m. ET on Dec 12.