FDA Grants Accelerated Approval To Sarepta' Drug For Duchenne Muscular Dystrophy

(RTTNews) - Sarepta Therapeutics Inc. (SRPT) said that the U.S. Food and Drug Administration has granted accelerated approval for EXONDYS 51 or eteplirsen as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy or DMD in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

SRPT closed Monday's regular trading at $48.94, up $20.79 or 73.85 percent. In the after-hours, the stock further gained $1.21 or 2.47 percent.

The indication is based on an increase in dystrophin in skeletal muscles observed in some patients treated with EXONDYS 51. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Sarepta plans for a US Commercial launch to commence immediately.

The most common adverse reactions compared to a placebo group were vomiting (38%) and balance disorder (38%) with contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection also reported more frequently than placebo (= 10%).

The underlying cause of Duchenne muscular dystrophy is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Certain genetic mutations in DMD involve the deletion of exons, which interrupt proper translation of the genetic code into protein.

Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder affecting an estimated one in approximately every 3,500 - 5,000 males born worldwide. It is estimated that up to thirteen percent of people with DMD have mutations addressable by EXONDYS 51.