- The FDA has granted its much-sought priority review status to Sarepta Therapeutics' eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD).
- DMD is a rare genetic disease, which mainly affects young men. Because the body does not produce dystrophin in those affected by DMD, muscle tissue does not function properly, the body weakens and eventually the patient is paralyzed and dies. Most patients die before age 30.
- Eteplirsen has previously received fast-track status, orphan drug designation and rare pediatric disease designation.
Both Sarepta and Biomarin, which is developing drisapersen for treatment of DMD, have benefited from recent guidance released by the FDA on June 10 outlining key aspects of clinical trial design for DMD candidates. The guidance, which reflects FDA flexibility with respect to trial size and other factors, came about after physicians, patients and DMD advocates pushed for clinical trials to better reflect the reality of DMD and the size of the affected population—after a period in which the FDA had rejected Sarepta's application because the agency felt that the trial was too small.
Another likely important factor is Sarepta's progress with eteplirsen is the company's new advisor—Henri Termeer, former CEO of Genzyme. Termeer's role has been to help Sarepta with the eteplirsen application. Given the fact that Termeer came on board only last month, it seems that his presence has been helpful—and that Sarepta has finally progressed to the point where an approval is in sight.