The US Food and Drug Administration (FDA) has informed Sarepta Therapeutics that it recommends resuming shipments of its gene therapy, ELEVIDYS, to ambulatory patients with Duchenne muscular dystrophy. The decision follows a comprehensive review of available safety information by the FDA.
Sarepta will resume shipping ELEVIDYS to sites of care for treatment of ambulatory patients with Duchenne imminently. The company commends the FDA for its swift and comprehensive review of available data, which included the case of an 8-year-old patient with a confirmed mutation in the DMD gene.
ELEVIDYS is approved for use in individuals at least 4 years old with a confirmed mutation in the DMD gene. However, patients who are non-ambulatory and have this condition require accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle.
The FDA has also warned of potential risks associated with ELEVIDYS treatment, including infusion-related reactions, acute serious liver injury, immune-mediated myositis, myocarditis, and preexisting immunity against AAVrh74. Patients must be closely monitored for these conditions and may require additional treatment.
Sarepta remains committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients.
Source: https://investorrelations.sarepta.com/news-releases/news-release-details/fda-informs-sarepta-it-recommends-sarepta-remove-its-pause-and