By Fineline Cube 
US-based Sarepta Therapeutics, a subsidiary of Sanofi (NASDAQ: SNY), announced the layoff of approximately 500 employees—about 36% of its workforce—and a restructuring of its R&D pipeline. The company will now focus on its siRNA platform for developing therapies in neurodegenerative and other disease areas. This decision follows the FDA’s suspension of clinical trials related to Sarepta’s gene therapy product, Elevidys.
FDA actions
According to an FDA statement, three non-ambulatory patients with Duchenne muscular dystrophy (DMD) died after receiving Sarepta’s gene therapies based on its AAVrh74 platform. One of these deaths was linked to Elevidys and involved acute liver failure. Consequently, the FDA has ordered Sarepta to suspend the distribution of Elevidys and all clinical trials involving the AAVrh74 platform. The FDA has also revoked the platform’s designation as a “platform technology.”
Impact on Elevidys
Elevidys received accelerated approval from the FDA in 2023 for non-ambulatory DMD patients. In 2024, the approved indication was expanded to include ambulatory patients aged four years and older. In response to the safety events, the FDA has required Sarepta to update its product labeling with a boxed warning and to limit the approved indication to ambulatory DMD patients aged four and above.
Future Direction
While Sarepta will continue advancing SRP-9003 (for LGMD-2E), several other programs, including most of its gene therapy candidates for limb-girdle muscular dystrophy (LGMD), will be paused. Sarepta stated that these measures aim to reduce operating costs, optimize resource allocation, and realign its R&D strategy amid changing regulatory conditions.-Fineline Info & Tech