FDA finally grants approval for Exondys 51, first drug for Duchenne MD

After many delays and calls for additional data, the US Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD), from US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT). The news saw Sarepta’s leap more than 90% to $53.56 by midday today (Monday).

Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD. An FDA advisory panel had previously voted against approval of the drug, as well as others for the treatment of DMD, amid strong protests from patient groups.

“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval,” she explained.