Health
FDA Clears First Gene Therapy for Kids’ Deadly Muscle Disease
- FDA cleared Elevidys only for patients between ages of 4 and 5
- Sarepta will charge $3.2 million per patient for the treatment
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Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy secured accelerated approval in the US for some children with the lethal, muscle-wasting disease.
The Food and Drug Administration cleared the treatment for patients only between the ages of 4 and 5, the group that saw the biggest benefit in clinical trials, the company said in a statement Thursday. While the decision strikes a middle ground after questions arose about the treatment’s effectiveness, parents of older children will likely oppose the limitation.