The United States has imposed significant restrictions on the gene therapy treatment known as Elevidys, following the deaths of two adolescent patients from acute liver failure after receiving the infusion. The Food and Drug Administration (FDA) announced on October 6, 2023, that the treatment will now be limited to children aged four and older who are able to walk, marking a substantial narrowing of its approved use.
Both patients who died had been diagnosed with Duchenne muscular dystrophy, a severe genetic disorder that leads to progressive muscle degeneration. Prior to treatment, they had lost the ability to walk, underscoring the critical need for vigilance in administering this therapy. The average life expectancy for individuals with Duchenne muscular dystrophy is just 28 years, as the disease often leads to complications affecting the heart and respiratory systems.
The FDA’s recent decision follows a series of actions taken over the summer. In July, the agency suspended clinical trials of Elevidys and initiated a safety review. At that time, it requested Sarepta, the biopharmaceutical company that developed the therapy, to halt all shipments of the drug to non-ambulatory patients. The company has since stated that it will continue to provide Elevidys to patients who meet the new criteria.
European regulators have also scrutinized Elevidys. The Committee for Medicinal Products for Human Use (CHMP) recommended against marketing approval for the therapy in July, citing insufficient data to demonstrate its efficacy in improving movement abilities. This recommendation highlights the ongoing debate surrounding the treatment’s safety and effectiveness, which has reportedly created tensions within the FDA as families and advocacy groups seek viable options for managing this debilitating condition.
To mitigate risks, the FDA’s new labeling for Elevidys will include recommendations for close monitoring of liver function during the first three months of treatment. Specifically, it will advise weekly assessments to ensure patient safety. These measures reflect the urgency of addressing the serious side effects associated with the therapy while also acknowledging the desperate need for effective treatments for Duchenne muscular dystrophy.
As discussions continue about the future of Elevidys and similar therapies, the focus remains on balancing patient safety with the imperative to provide effective medical options for those affected by this life-altering disease.
