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FDA Expands Approval of Gene Therapy for Patients with Muscular Dystrophy
The FDA expanded the approval of Elevidys, a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years
In making this decision, the FDA considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact. The most commonly reported side effects by individuals who received Elevidys were vomiting, nausea, acute liver injury, fever and thrombocytopenia (abnormally low platelet count in the blood).
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