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. “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. 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.

Source: CNN June 26, 2023 17:51 UTC