Sarepta Therapeutics encounters a setback as the Food and Drug Administration prepares to probe the demise of a child following the administration of Elevidy, their drug.
In a recent development, the U.S. Food and Drug Administration (FDA) is actively investigating the death of an 8-year-old boy who received Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics. The boy passed away on June 7, 2025. Elevidys is an adeno-associated virus (AAVrh74) vector-based gene therapy designed to treat this genetic disorder characterized by progressive muscle weakening[1].
This investigation follows three other patient deaths – two teenage boys and a 51-year-old man – who died after receiving Elevidys or a related Sarepta gene therapy for limb girdle muscular dystrophy. The FDA has requested a voluntary suspension of Elevidys distribution to investigate safety concerns, primarily related to acute liver failure[1][2][3][5].
Initially, Sarepta Therapeutics, a company listed on NASDAQ with the ticker symbol SRPT, resisted the request to halt all shipments but eventually agreed to voluntarily and temporarily pause Elevidys shipments for all DMD patients, acknowledging the seriousness of these safety signals despite the treatment's potential benefits[3].
The investigation centres on the safety of the AAVrh74 viral vector platform used by Elevidys, as the FDA revoked Sarepta's platform technology designation because early evidence failed to demonstrate safe applicability beyond a single drug[5]. These deaths have raised concerns about liver toxicity and acute liver failure linked to this gene therapy or investigational therapies using the same vector[2][5].
Roche Holding AG, a partner in the development of Elevidys, stated that a recent death of a patient in Brazil who received the therapy was unrelated to it, which may indicate ongoing investigation into causality[4].
Crucially, the FDA has indicated that continued approval of Elevidys for non-ambulatory patients (those who cannot walk) depends on verified clinical benefit through confirmatory trials, and due to new safety information, the agency recommends restricting Elevidys use to ambulatory patients only[5].
This ongoing investigation is significant as it highlights the need for continued monitoring and evaluation of gene therapies in the treatment of Duchenne muscular dystrophy and related disorders. The FDA, Sarepta, and Roche are closely monitoring safety data to ensure patient protection.
It is essential to note that the text did not provide information on the outcome of the new studies that the FDA has required Sarepta Therapeutics to conduct.
References:
- FDA investigates Sarepta's gene therapy Elevidys after patient death
- FDA investigates Sarepta's gene therapy Elevidys following patient deaths
- Sarepta pauses shipment of gene therapy Elevidys following FDA request
- Roche says death of Elevidys patient in Brazil not linked to therapy
- FDA places clinical hold on Sarepta's gene therapy trials
- The ongoing investigation by the FDA into Sarepta Therapeutics' gene therapy Elevidys, used for treating chronic-kidney-disease associated medical-conditions like Duchenne muscular dystrophy, has revealed safety concerns, primarily linked to acute liver failure.
- The stock-market listed company, Sarepta Therapeutics, has agreed to voluntarily pause Elevidys shipments, acknowledging the seriousness of the safety signals, despite the drug's potential benefits, following the FDA's request.
- The deaths of several patients who received Elevidys or a related Sarepta gene therapy for limb girdle muscular dystrophy have raised concerns about chronic-diseases related to liver toxicity and acute liver failure connected to this gene therapy or investigational therapies using the same vector.
- In light of the new safety information, the FDA has recommended restricting the use of Elevidys to ambulatory patients only, but continued approval for non-ambulatory patients depends on verified clinical benefit through confirmatory trials.
