The United States has announced significant changes to the regulation of a gene therapy aimed at treating Duchenne muscular dystrophy, a severe muscle-wasting disease. The Food and Drug Administration (FDA) will now apply its most serious warning label to Elevidys and limit its approved use following the deaths of two adolescent patients from acute liver failure after receiving the treatment.
Both patients were non-ambulatory at the time they began the therapy. As a result of this decision, the FDA will restrict the prescription of Elevidys to individuals who can walk and are older than four years. Duchenne muscular dystrophy is a rare and fatal condition predominantly affecting boys, typically leading to significant muscular deterioration, including impairment of the heart. The average life expectancy for those diagnosed with this condition is approximately 28 years.
In July 2023, Sarepta Therapeutics, the biopharmaceutical company that developed Elevidys, paused distribution of the treatment for non-ambulatory patients. This followed the FDA’s announcement to halt clinical trials and initiate a safety review of the therapy. The agency also requested Sarepta to cease all shipments of Elevidys, although the company indicated it would continue to provide the treatment to patients who can walk.
Regulatory Changes and Safety Precautions
The FDA’s new guidelines will include recommendations for patients to undergo weekly liver function monitoring during the first three months of treatment. This precautionary measure aims to mitigate risks associated with the therapy.
Meanwhile, the European Committee for Medicinal Products for Human Use recommended against the marketing approval of Elevidys, citing insufficient evidence to demonstrate the treatment’s effectiveness in improving movement abilities. The debate surrounding the approval of Elevidys has reportedly caused internal tensions within the FDA, especially as advocacy groups and parents of affected children continue to seek viable treatment options.
Despite these challenges, the push for effective therapies remains a priority for many families. The regulatory changes underscore the complexity of balancing treatment options with patient safety in the face of a devastating disease.
