Sarepta suspends non-ambulatory Elevidys shipments after second death

Sarepta (SRPT) Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular dystrophy. The company announced a second reported case of acute liver failure resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, remains the same. Sarepta has also voluntarily paused dosing in the ENVISION clinical study. FDA concurs with this action, the company said. “The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume,” Sarepta added. The company is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys.