Third Sarepta Patient Dies from Gene Therapy

Rare Daily Staff

A Sarepta Therapeutics patient died from acute liver failure following treatment with the company’s experimental gene therapy for limb-girdle muscular dystrophy, Bloomberg reported.

The company told Bloomberg that the death had been reported “in an appropriate and timely manner” to regulators and investigators. The case followed the death of two non-ambulatory boys from acute liver failure following treatment with the company’s gene therapy for Duchenne muscular dystrophy.

Earlier this week, the company announced a reorganization that included cutting about one-third of its staff and a focus on its siRNA programs in development. It also said the U.S. Food and Drug Administration requested that the label for its Duchenne gene therapy Elevidys include a black box warning for acute liver injury and acute liver failure. Sarepta said it agrees with this change, which it believes resolves any material issues with the ambulatory portion of the Elevidys label.

It also described an enhanced immunosuppressive regimen with sirolimus for Elevidys in non-ambulatory patients that a committee of experts it had convened recommended. Dosing in non-ambulatory patients had previously been paused. Sarepta is submitting the findings of the panel and proposed protocol to the FDA and said it will discuss a proposal to gather data on the regimen in a new cohort of its ENDEAVOR study as a pathway to re-establish dosing in the non-ambulatory setting.