Sarepta Therapeutics has announced a strategic restructuring plan designed to prioritise high-value, high impact programmes, and support its long-term financial viability.  

This plan includes a 36% workforce reduction, impacting approximately 500 employees, and an emphasis on near- and mid-term opportunities from the siRNA platform. 

Several programmes, including most of the gene therapies in development for limb-girdle muscular dystrophy (LGMD), will be paused.  

Although Sarepta plans to submit the Biologics License Application for SRP-9003 for LGMD type 2E/R4 in the second half of 2025, it intends to seek strategic alternatives, including partnering, for programmes that it no longer intends to fund directly. 

Collectively, the measures are projected to deliver approximately $400 million in annual cost reductions. 

“Faced with environmental changes, we have decided to act decisively, implementing a focused strategy to ensure Sarepta remains a vibrant, financially enduring, patient-centric organisation dedicated to improving the lives of those with rare genetic diseases,” said Doug Ingram, Chief Executive Officer, Sarepta Therapeutics. “We will continue to drive performance of Elevidys and our three PMOs in service of the Duchenne community those therapies benefit, and with our financial performance, we will advance our high-value, focused pipeline of programmes for rare genetic diseases, primarily relying on the siRNA platform, while ensuring we meet our financial obligations.” 

Following the deaths of two non‑ambulatory Duchenne muscular dystrophy (DMD) patients and in response to a request from the FDA, the company has added a black box warning to the Elevidys (delandistrogene moxeparvovec) label for acute liver injury (ALI) and acute liver failure (ALF). 

Sarepta recently convened a committee of specialists to review cases of ALF, which recommended an enhanced immunosuppressive regimen with sirolimus for Elevidys in non-ambulant patients. 

The company says it is also assessing real-world data generation opportunities for ambulant patients through investigator-initiated trials.

Diana Turner, Senior Digital Content Editor, DDW