Fact checked byHeather Biele

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June 23, 2023
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FDA approves Elevidys for pediatric Duchenne muscular dystrophy

Fact checked byHeather Biele
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The FDA has approved Elevidys, a recombinant gene therapy for pediatric patients with Duchenne muscular dystrophy and confirmed gene mutation who do not have a pre-existing medical reason preventing treatment.

According to an FDA release, Elevidys (delandistrogene moxeparvovec-roki, Sarepta Therapeutics) delivers a gene into the body that produces micro-dystrophin, a shortened protein containing selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single IV dose in patients aged 4 to 5 years.

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The FDA has approved single IV dose Elevidys for pediatric Duchenne muscular dystrophy. Image: Adobe Stock

Elevidys was approved through the FDA’s accelerated approval pathway, based on data evaluation of a randomized, double-blind, placebo-controlled clinical trial submitted by Sarepta Therapeutics, the release stated.

The two-part study, in which participants were treated with Elevidys or placebo for 48 weeks and switched therapies for an additional 48 weeks, established that Elevidys increased expression of the micro-dystrophin protein observed in Elevidys-treated individuals aged 4 to 5 years with Duchenne muscular dystrophy (DMD).

The FDA concluded that the data submitted by Sarepta demonstrated that an increase in the expression of micro-dystrophin is likely to provide clinical benefit to the target patient group who do not have significant pre-existing antibody titers against the AAV rh74 vector or other contraindications, based on inclusion criteria of the clinical trials.

“Today’s approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual’s health over time,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the release. “The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”

As a condition of approval, the FDA will require Sarepta to complete a clinical study — which is ongoing and fully enrolled — that confirms Elevidys’ efficacy in improving physical function and mobility in ambulatory DMD patients with a confirmed mutation in the DMD gene.