FDA Approves $3.2 Million Gene Therapy for Fatal Childhood Disease Despite Research Concerns

FDA approved the first gene therapy treatment for Duchenne muscular dystrophy, a rare muscle-wasting disease that causes severe disability and early death, in children ages 4 and 5.
The gene therapy, called Elevidys, provides a one-time infusion of a working gene to replace one that leads to disease.
The therapy costs $3.2 million per patient and must show in an ongoing clinical trial that it improves mobility and physical function in patients to stay on the market.
FDA scientists questioned the treatment's benefits and potential safety risks, but outside experts narrowly voted to approve the therapy.
Patient advocates argue that drugs offering even modest improvements can help bridge the gap until better therapies emerge.