The United States has announced significant changes to the use of a gene therapy treatment known as Elevidys, aimed at combating the debilitating effects of Duchenne muscular dystrophy. The Food and Drug Administration (FDA) is adding a serious warning label and restricting its approved usage following the tragic deaths of two adolescent patients due to acute liver failure after receiving the treatment.
Both patients were already non-ambulatory at the time they began the therapy. The FDA’s decision means that Elevidys will now only be prescribed to patients who can still walk and are older than four years. This condition, which predominantly affects boys, leads to severe muscle degeneration, including potential heart failure, and has a grim average life expectancy of just 28 years.
In the summer of 2023, Sarepta Therapeutics, the biopharmaceutical company behind Elevidys, temporarily halted distribution to non-ambulatory patients. This pause followed the FDA’s decision in July to suspend trials and initiate a safety review of the therapy. The agency also requested that Sarepta cease all shipments of Elevidys until further notice.
The situation has drawn attention not only in the United States but also in Europe. In July, the Committee for Medicinal Products for Human Use recommended against granting marketing approval for Elevidys, citing insufficient data to demonstrate the treatment’s effectiveness in improving mobility.
The FDA’s revised guidelines will include recommendations for necessary precautions, such as weekly liver function monitoring for the first three months after the initiation of treatment. The agency’s actions reflect ongoing concerns regarding the safety and efficacy of this therapy, amid a backdrop of increasing pressure from advocacy groups seeking effective treatment options for patients suffering from Duchenne muscular dystrophy.
Debate surrounding the approval of Elevidys has reportedly created tension within the FDA, highlighting the complexities involved in balancing patient needs with safety considerations. The agency’s new restrictions aim to ensure that the potential benefits of the treatment are weighed against the risks, particularly in vulnerable patient populations.
As the situation develops, both the FDA and Sarepta remain committed to monitoring patient safety and ensuring that those who can benefit from Elevidys receive it in a managed and responsible manner.
