Sarepta Therapeutics shares spiked by as much as 86% in trading on Monday after the US Food and Drug Administration said it approved a key drug.
The FDA approved Exondys 51 (eteplirsen), the first drug approved to treat patients with Duchenne muscular dystrophy (DMD), it said in a statement.
The disease is a rare disorder that affects one in every 3,500 to 5,000 males worldwide. It’s caused by the absence of dystrophin, a protein that helps sustain muscles, and gradually but severely damages muscles.
Patients could need wheelchairs even in their early teens, and can cause death by the time they are in their 30s.
“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,” said Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, in the statement.
Sarepta shares were halted after the spike.
More to come …