FDA OKs Elevidys As First Gene Therapy To Treat Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. announced Friday that the U.S. Food and Drug Administration or FDA has accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

DMD is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. It is a relentlessly progressive, degenerative disease that occurs due to a defective gene that results in absence of dystrophin, a protein that helps keep the body’s muscle cells intact.

Although disease severity and life expectancy vary, patients often succumb to the disease in their 20s or 30s because of heart and/or respiratory failure.

Most current treatment approaches address the symptoms of the disease, but not its underlying genetic cause. The treatments include corticosteroid medications to slow down the progression of muscle weakness.

ELEVIDYS is a one-time treatment designed to treat the underlying genetic cause of DMD. This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. The continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

ELEVIDYS, an adeno-associated virus based gene therapy, addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin.

ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. This accelerated approval is based on an increase in ELEVIDYS micro-dystrophin protein expression in skeletal muscle.

ELEVIDYS is supported by biologic and empirical evidence, in addition to efficacy data from two clinical studies: SRP-9001-102 and SRP-9001-103 and safety data from SRP-9001-101, SRP-9001-102 and SRP-9001-103.

Amid the accelerated approval pathway, the company has committed to the completion of a confirmatory trial EMBARK, which is a global, randomized, double-blind, placebo-controlled Phase 3 trial for ELEVIDYS. This trial will serve as the post-marketing confirmatory trial and is fully enrolled with top-line results expected in late 2023.

In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease for these children, and the urgent unmet medical need.

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