In a recent press release, Sarepta Therapeutics outlined the steps they are taking to ensure the safety of Elevidys® (delandistrogene moxeparvovec-rokl), a gene therapy for patients with Duchenne muscular dystrophy (DMD). The announcement comes after a second fatal case of acute liver failure was reported following drug administration.
“Sarepta extends its deepest sympathies to the affected families and care teams,” the company wrote.
Both deaths occurred in non-ambulatory patients, or individuals who are no longer able to walk without assistance. Although increased levels of liver enzymes are not uncommon with this class of therapy, the mechanisms of liver toxicity and acute liver failure are still unknown.
Elevidys was approved by the U.S. Food and Drug Administration (FDA) in June 2023 and remains the only FDA-approved gene therapy for patients with DMD. It is currently available to patients who are four years of age and older.
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Sarepta is working with a panel of experts in DMD and liver health to determine how they may prevent additional cases of liver failure in the future. As part of their plans, they expect to modify the immunosuppressive regimen that is given to patients prior to Elevidys administration to reduce the risk of immune response.
While these discussions are occurring, Sarepta has decided to temporarily suspend all shipments of Elevidys to non-ambulatory patients. No changes will be made for ambulatory patients at this time.
Additionally, Sarepta is temporarily pausing their Phase 3 ENVISION clinical trial, which is required for accelerated approval for non-ambulatory patients. The study aims to evaluate the efficacy of Elevidys in non-ambulatory individuals and in ambulatory individuals aged eight to 18 years.
The company will share the expert panel’s suggestions with the FDA before making amendments to their treatment regimen. Updates will also be shared with the general public.
“With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients,” said Dr. Louise Rodino-Klapac, chief scientific officer and head of research and development at Sarepta.
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