FDA Grants Accelerated Approval to Elevidys for Duchenne Muscular Dystrophy

The FDA has granted accelerated approval delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics Inc), making it the first gene therapy approved for the treatment of pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have pre-existing medical conditions that prevent treatment with this therapy.¹

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“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 a statement. “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.”

Duchenne muscular dystrophy is a rare generic condition, which worsens over time, according to the statement. It causes the body’s muscles to weaken and waste away. Symptoms can include trouble walking and running, falling frequently, fatigue, learning disabilities or difficulties, heart problems, and breathing problems.¹

Delandistrogene moxeparvovec-rokl is a recombinant gene therapy that delivers to the body a gene that leads to the production of micro-dystrophin, a shortened protein that select domains of the dystrophin protein present in the normal muscle cells. The therapy is administered as a single intravenous dose.¹

The approval was based primarily on data from 2 studies. Study 1 is ongoing, with part 1 including a 48-week, randomized, double-blinded, placebo-controlled period and part 2 including a 48-week period that began after the completion of part 1 with patients who received the placebo in part 1 being treated with delandistrogene moxeparvovec-rokl and those treated with delandistrogene moxeparvovec-rokl receiving the placebo.²

Investigators found that in the total North Star Ambulatory Assessment (NSAA), there was no statistically significant difference between the therapy and the placebo group. However, in the subgroup analyses, investigators found that for individuals aged 4 through 5 years, there was a numerical advantage to delandistrogene moxeparvovec-rokl for the least squares mean change in NSAA total score.2

The accelerated approval was based on data from a randomized clinical trial that evaluated the increased expression of micro-dystrophin proteins observed in those treated with delandistrogene moxeparvovec-rokl, according to the FDA.¹

A clinical benefit of delandistrogene moxeparvovec-rokl has not yet been seen, which includes improved motor function, according to the FDA. As part of the approval condition, the FDA is requiring Sarepta Therapeutics to complete a clinical study to confirm the drug’s benefit.¹

The required study is meant to assess whether the therapy improved physical function and mobility for individuals with ambulatory DMD who have the confirmed mutation.¹

References

1. FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy. News release. US Food and Drug Administration. June 22, 2023. Accessed June 22, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy?utm_medium=email&utm_source=govdelivery
2. Elevidys (delandistrogene moxeparvovec-rokl. Prescribing Information. US Food and Drug Administration. Accessed June 22, 2023. https://www.fda.gov/media/169679/download