Thursday, the FDA approved labeled indication for Sarepta Therapeutics Inc's (NASDAQ:SRPT) Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.
Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients (patients who can walk) and accelerated approval for non-ambulatory patients (patients who cannot walk).
Related: Sarepta Downgraded As FDA Decision Date Approaches, Shares Up 50% Over Last Six Months.
Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
In June last year, the FDA granted accelerated approval to Sarepta Therapeutics's one-time gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients aged 4 through 5 with a confirmed mutation in the DMD gene.
Consistent with the accelerated approval pathway, Sarepta will conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of Elevidys in non-ambulatory patients with Duchenne muscular dystrophy.
ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement.
Sarepta is working with Roche Holdings AG (OTC:RHHBY) as part of a collaboration agreement signed in 2019.
Sarepta is responsible for regulatory approval, manufacturing, and commercialization of Elevidys in the U.S.
Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.
Last week, Pfizer Inc (NYSE:PFE) disclosed disappointing results from its Phase 3 CIFFREO study, evaluating the investigational gene therapy for Duchenne muscular dystrophy (DMD) in young boys.
The study did not achieve its primary endpoint of improving motor function in patients aged 4 to 7 years.
Also Read: Second Death – Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial.
CNN reports that Elevidys costs around $3.2 million per patient, making it the second most expensive drug globally, after the $3.5 million hemophilia treatment Hemgenix.
Price Action: SRPT shares are up 37.6% at $169.98 during the premarket session at last check Friday.
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