Sarepta Therapeutics, Inc. (NASDAQ:SRPT) has seen its stock price surge by more than 11% in premarket trading after the U.S. Food and Drug Administration (FDA) accepted and filed its Efficacy Supplement application to the Biologics License Application (BLA) for ELEVIDYS.
The Efficacy Supplement serves two main purposes: firstly, to broaden ELEVIDYS's use for treating Duchenne muscular dystrophy (DMD) in patients verified to have a mutation in the DMD gene; and secondly, to elevate the drug's status from accelerated to traditional approval.
The FDA has assigned the application a Priority Review, setting a target decision date of June 21, 2024, and has decided against convening an advisory committee meeting on the matter.
"We are pleased to announce that FDA has accepted and filed Sarepta’s Efficacy Supplement to evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the approval from accelerated to traditional,” said Doug Ingram, president and CEO of Sarepta Therapeutics.
“Understanding that every day matters to families living with Duchenne, we will work with our regulatory counterparts to successfully complete this review as rapidly as possible."