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 green-lighted Exondys 51 (eteplirsen), the first approved drug to treat patients with Duchenne muscular dystrophy (DMD), it said in a statement.
DMD is a rare disorder in males caused by the absence of dystrophin, a protein that helps keep muscle cells intact.
It causes gradual but severe damage while limiting movement. Patients could need wheelchairs in their early teens, and could die by the time they are in their 30s.
“The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in some patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping,” the statement said.
The FDA confirmed last week that Ronald Farkas, a staffer who opposed the drug partly because of its small 12-patient clinical trial, had left the agency, Stat News reported. Sarepta shares soared then, as Farkas’ departure was seen by investors to pave the way for approval.
Advocacy groups had pressured the FDA to endorse the drug, following the rejection of two treatments from other companies, Stat News noted.
“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 briefly halted for volatility after the spike on Monday.