FDA says Sarepta can resume Elevidys use in ambulatory patients

The U.S. FDA notified Sarepta Therapeutics that it may lift its voluntary pause on shipments of Elevidys for ambulatory patients with Duchenne, the company said in a press release.

Sarepta will resume shipping Elevidys to sites of care for treatment of ambulatory patients imminently.

Last week, Sarepta said it would voluntarily and temporarily pause all shipments of the gene therapy in the U.S., following a public dispute with the FDA.  

“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, Sarepta CEO.

The agency’s review of the safety data in the ambulatory population included the case of an 8-year-old in Brazil. According to Sarepta, FDA’s investigation concluded the death was unrelated to treatment with Elevidys.

The pause on shipments of Elevidys for non-ambulant patients — announced last month —  remains in place while the company works with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure. The decision was made following the second non-ambulatory patient death following treatment with Elevidys.