Sarepta Therapeutics has yielded to the US Food and Drug Administration (FDA), agreeing to suspend shipments of Elevidys (delandistrogene moxeparvovec) to the US.
On July 17, the FDA requested Sarepta pause shipments of the Duchenne muscular dystrophy (DMD) gene therapy, but the biopharmaceutical company initially declined, stating that there had been no new safety signals identified.
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On July 22, Sarepta agreed to the voluntary pause in shipments, with CEO Doug Ingram saying that it is important the company maintains a “positive working relationship” with the agency.
Ingram said: “As a patient-centric organisation, the decision to voluntarily and temporarily pause shipments of Elevidys was a painful one, as individuals with DMD are losing muscle daily and in need of disease-modifying options.
“It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process.”
Sarepta confirmed it will work with the FDA to respond to requests for information while advancing the Elevidys safety labelling supplement process.
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By GlobalDataThis toe-to-toe comes after a third patient death from a Sarepta gene therapy was revealed on 17 July. A 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 died after receiving Sarepta’s gene therapy candidate SRP-9004 in a Phase I trial. The patient’s death was revealed by the media and announced to investors the following day.
In the same statement requesting that Sarepta pause its Elevidys shipments, the FDA also put a clinical hold on studies of SRP-9004.
This comes after two deaths were announced following treatment with Sarepta’s Elevidys. Both patient deaths, the first of which was announced in March 2025 and the second in June 2025, were due to acute liver failure (ALF).
Elevidys and SRP-9004 both use the same recombinant adeno-associated viral vector (AAV) in AAVrh74.
In what has been a turbulent few days for Sarepta, the company recently said it was cutting 500 jobs as part of its restructuring and pipeline prioritisation plan, as well as updating the label for Elevidys. The company is engaging with the FDA regarding the label update for the gene therapy, which includes adding a black box warning for acute liver injury (ALI) and ALF.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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