Sarepta Therapeutics has announced a voluntary pause of ELEVIDYS shipments in the US due to safety concerns. The company is taking this proactive step to address any requests for information and complete the safety labeling supplement process with the FDA.
ELEVIDYS, a single-dose gene transfer therapy for Duchenne muscular dystrophy, was approved by the FDA in 2021. However, recent clinical trial data has raised concerns about infusion-related reactions, acute serious liver injury, immune-mediated myositis, myocarditis, and preexisting immunity against AAVrh74.
The company remains committed to transparency and patient safety, and will provide timely updates on ELEVIDYS as additional information becomes available. Sarepta is also working closely with the FDA to review its safety labeling supplement process.
Duchenne muscular dystrophy is a genetic disorder that affects approximately 1 in 5,000 male births. The disease causes progressive muscle degeneration, leading to loss of muscle function and mobility. ELEVIDYS is designed to address this underlying cause by delivering a transgene that codes for targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
The FDA has warned about potential risks associated with ELEVIDYS, including infusion-related reactions, liver injury, immune-mediated myositis, myocarditis, and preexisting immunity against AAVrh74. Patients who have received ELEVIDYS are advised to contact their healthcare provider if they experience any unusual symptoms or side effects.
Sarepta is working to address these concerns and provide a safe and effective treatment option for patients with Duchenne muscular dystrophy.
Source: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys