On March 19, 2025, Sarepta Therapeutics reported that a patient died while undergoing treatment with its gene therapy, Elevidys, for muscular dystrophy. The young man experienced acute liver injury, a known side effect of the therapy, which has been administered to over 800 patients, marking the first death associated with Elevidys.
- Patient death linked to Elevidys therapy
- Company shares dropped over 25%
- Acute liver injury identified as side effect
- Elevidys approved for Duchenne muscular dystrophy
- Cost of Elevidys is $3.2 million
- Ongoing studies for full FDA approval
Elevidys, which utilizes a disabled virus to deliver a replacement gene for dystrophin production, received expedited approval from the FDA in 2023 despite concerns about its effectiveness. The therapy is priced at $3.2 million for a one-time infusion and is specifically designed for patients with Duchenne muscular dystrophy, a severe condition that primarily affects males and leads to muscle weakness and loss of mobility.
Following the patient’s death, Sarepta indicated that a recent infection might have contributed to the liver injury. The company plans to update the prescribing information for Elevidys to include this critical case. In addition to Elevidys, Sarepta has received FDA accelerated approval for three other Duchenne’s drugs since 2016, although none have yet been confirmed effective, with studies ongoing to secure full approval.
After the announcement, Sarepta’s shares fell more than 27%, closing at approximately $73 each. This incident raises significant concerns regarding the safety of gene therapies, particularly in light of the ongoing debates about their efficacy and the regulatory processes involved in their approval.
The recent patient death linked to Elevidys underscores the complexities and risks associated with innovative gene therapies. As the situation develops, it will be crucial for Sarepta and regulatory agencies to ensure patient safety and transparency regarding treatment risks.
