Sarepta Therapeutics Soars on Expanded Approval for Its Muscular Dystrophy Drug

Sarepta Therapeutics (SRPT) shares soared in premarket trading Friday, a day after the Food and Drug Administration (FDA) allowed expanded use of the biotech’s treatment for a rare muscle disease in children, which especially affects boys.

The company said its drug, Elevidys, was given traditional approval for patients who are at least 4 years old and suffer from Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene and can walk.

Phase 3 Trial for Non-Ambulatory Individuals With DMD Currently Underway

Sarepta said regulators granted accelerated approval for use in those who cannot walk, and continued approval “may be contingent upon verification of clinical benefit in a confirmatory trial.” The company added that a Phase 3 study to make that confirmation is currently underway.

The FDA gave accelerated approval for the gene-therapy medicine in 4- and 5-year-olds last year, despite some questions about its efficacy. 

Dr. Jerry Mendell, co-inventor of Elevidys and a senior adviser at Sarepta, said that the initial approval was a significant milestone, “and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne.”

Sarepta shares soared 35% to $167.00 an hour before the opening bell Friday after closing Thursday up 5.1% to $123.50.