Sarepta Therapeutics (SRPT) shares soared in premarket trading Friday, a day after the 澳洲幸运5官方开奖结果体彩网: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 Tr𓄧ial for Non-Ambulatory Individuals With DMD Currently Underway

Sarepta said regulators grant💎ed accelerated approval for use in those who cannot walk, and continued approval “may be co🔯ntingent 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 ꦦefficacy. 

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 be𒊎ll Friday after closing Thursday up 5.1% to $123.50.