FDA Investigates Death Linked to Gene Therapy for Muscular Dystrophy

The U.S. Food and Drug Administration (FDA) has initiated an investigation into the death of an 8-year-old boy who received Elevidys, a gene therapy developed by Sarepta Therapeutics. The child, treated in Brazil, passed away on June 7, 2023. In response to safety concerns, the FDA has secured a voluntary suspension of Elevidys distribution in the United States while the case is evaluated.

Details on the Treatment and Safety Concerns

Elevidys is a one-time gene therapy administered via intravenous injection, aimed at assisting children with Duchenne muscular dystrophy, a rare genetic disorder that leads to progressive muscle weakness. According to the Muscular Dystrophy Association, the therapy utilizes a harmless virus to deliver a copy of the micro-dystrophin protein, which is crucial for muscle function in patients lacking the gene necessary for its production.

The boy’s death, while not part of a clinical trial, has been reported under the FDA’s post-marketing surveillance system. Despite this, Roche, the company responsible for distributing Elevidys outside the U.S., stated that the treating physician deemed the death unrelated to the therapy. Nevertheless, the FDA is proceeding with caution.

Previous Fatalities and Regulatory Response

This incident is particularly concerning as it marks the third fatality associated with Sarepta’s gene therapies in recent months. Reports indicate that two teenage boys died from acute liver failure following treatment, and a 51-year-old man receiving SRP-9004, an experimental therapy that employs the same viral vector as Elevidys, also died.

Initially, Sarepta resisted the FDA’s request to halt U.S. distribution of Elevidys. However, facing increasing regulatory pressure and a significant drop in its stock price, the company ultimately agreed to suspend shipments. The suspension is expected to last several months as the FDA continues to investigate.

The situation underscores the complexities and risks associated with gene therapy, highlighting the need for ongoing oversight and rigorous safety evaluations for emerging treatments.