Sarepta lays off 500 workers, agrees to black box warning for Elevidys

Sarepta Therapeutics announced a strategic restructuring plan designed to reduce operating expenses, with the aim of delivering $400 million in annual cost savings.

The plan includes a 36% workforce reduction of approximately 500 employees.

Sarepta also will reprioritize its pipeline to focus on “high-impact programs” — specifically its siRNA platform assets. The siRNA programs include investigational treatments for facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis and Huntington’s disease. This means that several programs, including most of the gene therapies in development for limb-girdle muscular dystrophy, will be paused.

In the same press release, Sarepta announced that, at the request of the FDA, the company will revise the Elevidys label to include a black box warning for acute liver injury and acute liver failure.

Last month, Sarepta revealed that was halting the commercial and clinical use of Elevidys gene therapy for non-ambulatory Duchenne muscular dystrophy patients, following a second case of fatal acute liver failure. The company also promised it would convene an independent group of experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys.

Now, Sarepta says the committee aligned on an enhanced immunosuppressive regimen with sirolimus for Elevidys in non-ambulant patients. Sarepta plans to submit the findings and proposed protocol to the FDA and propose a new study cohort as a pathway to re-establish dosing in the non-ambulant setting.