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Drug expected to support children aged 4 and 5 with Duchenne muscular dystrophy.
Sarepta Therapeutics, a drug development company, has announced the FDA-approval of Elevidys, a gene therapy designed to treat children with Duchenne muscular dystrophy. Approved under the accelerated pathway, the medication will reportedly need to prove that it improves physical function and mobility in an ongoing clinical trial.
Intended to be taken only once, Sarepta revealed the cost of the therapy will be $3.2 million per patient.
“Today’s approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual’s health over time,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected.”
Reference: FDA approves $3.2 million gene therapy for rare muscular dystrophy in kids ages 4 and 5. CNN. June 22, 2023. Accessed June 26, 2023. https://www.cnn.com/2023/06/22/health/fda-sarepta-muscular-dystrophy/index.html#:~:text=Duchenne%20muscular%20dystrophy%20causes%20progressive,cost%20%243.2%20million%20per%20patient.