Sarepta Therapeutics Inc (NASDAQ:SRPT) said the US Food and Drug Administration (FDA) has accelerated approval for ELEVIDYS, its first-of-a-kind gene therapy for Duchenne muscular dystrophy (DMD).
DMD is an inherited progressive muscle-wasting disorder that generally affects young boys. The company said the treatment has been approved for patients aged 4 through 5 years who can still walk.
The therapy's wholesale acquisition cost is $3.2 million, CEO Douglas Ingram said on a conference call with investors, according to a report by Reuters.
“The approval of ELEVIDYS is a watershed moment for the treatment of Duchenne," Ingram commented in a statement.
“ELEVIDYS is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease.”
Dr Jerry Mendell, pediatric neurologist and principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital added: “Duchenne is a relentlessly progressive, degenerative disease, robbing children of muscle function. The increases in ELEVIDYS dystrophin expression and the functional results that we see can make a difference in the lives of our patients.
The company said it has committed to the completion of a confirmatory trial under the accelerated approval pathway.
EMBARK, the global, randomized, double-blind, placebo-controlled Phase 3 trial for ELEVIDYS, will serve as the post-marketing confirmatory trial and is fully enrolled with top-line results expected in late 2023, it added.
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