Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has informed the company it may lift its voluntary pause on shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy (DMD). Sarepta will soon resume shipping the gene therapy to treatment centers for eligible patients.
The pause was initially implemented at the FDA’s suggestion to allow a thorough review of safety data. Following a rapid and comprehensive evaluation, the FDA concluded that ELEVIDYS shipments can resume safely for ambulatory individuals.
Doug Ingram, Sarepta’s CEO, expressed relief and optimism: “The FDA’s swift review demonstrates their commitment to the Duchenne community, a commitment we share. We look forward to collaborating on finalizing the safety label update and discussing risk mitigation strategies for non-ambulatory patients, who remain on pause pending further discussions.”
The FDA’s safety review considered, among other data, a case involving the death of an 8-year-old patient in Brazil. Brazilian health authorities determined the death was unlikely related to ELEVIDYS treatment, and the FDA’s investigation confirmed this finding.
ELEVIDYS remains the only approved gene therapy for Duchenne, a devastating and progressive rare disease. Sarepta reaffirmed its commitment to working closely with the FDA to ensure decisions prioritize patient safety and access to this important therapy.