Aggregated News
A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile.
Sarepta and its ex-U.S. partner Roche reported the death early Sunday. Like the first case, disclosed in March, the patient died after developing acute liver failure (ALF) and was non-ambulatory, meaning the individual was no longer able to walk independently because of the progression of Duchenne.
As a result, Sarepta has suspended giving Elevidys to non-ambulatory patients in the commercial setting in the U.S. while the company seeks FDA approval of an enhanced risk mitigation measure involving the immunosuppressant sirolimus to manage liver toxicity.
For its part, Roche said it has simply “discontinued” Elevidys in commercial non-ambulatory patients outside the U.S.
Meanwhile, Sarepta has paused dosing in a clinical trial called Envision, which is serving as the confirmatory trial required by the FDA for Elevidys’ accelerated approval in non-ambulatory patients. The Massachusetts biotech aims to amend the trial protocol to add the prophylactic use of sirolimus, CEO Doug Ingram said on a conference call Monday.
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