Shares of Sarepta Therapeutics (SRPT) - Get Report surged on Friday after the biotech company said it received accelerated Food and Drug Administration approval for its injectable treatment for a rare form of muscular dystrophy.
Shares of Sarepta Therapeutics jumped nearly 30%, rising $29.63 to $130.10 in morning trading on Friday after the company said it had received fast-track approval to roll out the drug, called Vyondys 53, that treats so-called Duchenne muscular dystrophy (DMD).
“With the approval of Vyondys 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease,” Parent Project Muscular Dystrophy CEO Pat Furlong said in a statement.
Sarepta noted a “statistically significant increase” in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53, which is reasonably likely to predict clinical benefit for those patients who are “exon 53-amenable,” a medical term for patients whose dystrophin genes mutated in response to the injected treatment.
Sarepta plans to begin U.S. distribution immediately. The company will continue with post-release confirmatory trials of the drug through 2024. Vyondys 53 is priced at parity to Exondys 51, the price of which has not increased since its launch in 2016, the company said.
Shares of Sarepta have gained more than 40% since October after the company announced positive results from a separate gene therapy trial to treat a different form of muscular dystrophy.
The company said the nine-month results showed improvements on functional measures seen in all three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants. Limb-girdle muscular dystrophy is a term for a group of diseases that cause weakness and wasting of the muscles in the arms and legs.