Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of a patient with Duchenne muscular dystrophy (DMD) following treatment with the marketed gene therapy. The patient, whose age was not disclosed, was a non-ambulatory individual with DMD. According to Sarepta, the patient was the second to succumb to acute liver failure (ALF) after being treated with Elevidys, which is the only gene therapy that has won FDA approval as a treatment for DMD. The first patient was a 16-year-old young man who died in March. At the time, Sarepta said it would update its prescribing information for Elevidys. “We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time,” Louise Rodino-Klapac, PhD, Sareota’s CSO and head of research & development, said in a statement. Sarepta said its temporary suspension of shipments would apply to non-ambulatory patients, and would remain in effect while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. No treatment changes will be implemented for ambulatory patients, Sareota added. “The current practice of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, remains the same.”

Full report : Sarepta reports second case of liver failure death after its gene therapy treatment.