Shipments of Elevidys, the only approved gene therapy for Duchenne muscular dystrophy (DMD), will resume immediately for ambulatory patients after the United States Food and Drug Administration (FDA) recommended Sarepta lift its voluntary pause, according to a press release published recently by Sarepta.
The company had halted distribution at the FDA’s request to allow time for a safety review. That evaluation is now complete, and the agency concluded the therapy can be delivered safely to individuals who are still able to walk.
The FDA’s recommendation followed the investigation of a reported death involving an 8-year-old patient in Brazil. Brazilian health authorities determined the child’s death was unlikely to be related to Elevidys, and the FDA’s own review confirmed no connection to the therapy. Based on these findings, the agency endorsed resuming shipments of Elevidys for ambulatory patients with DMD.
DMD is caused by mutations in the DMD gene, leading to the loss of dystrophin, a crucial muscle protein. Without it, muscle tissue weakens over time, often leaving patients unable to walk by their early teens and resulting in a shortened life span. Elevidys delivers a gene that helps the body produce a modified form of dystrophin.
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“We are very pleased that FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of ELEVIDYS for ambulatory patients. The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta,” said Doug Ingram, chief executive officer, Sarepta.
Ingram noted the urgency felt by families facing this devastating condition. The company said it would continue working with the FDA to finalize safety labeling updates and determine how to safely offer Elevidys to non-ambulatory patients, who remain on pause for now.
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