![]() ![]() The approval is the first ever for a gene therapy for Duchenne muscular dystrophy and Sarepta’s fourth overall treatment to be cleared for the disease. Shares of Sarepta were little changed in extended trading in New York. After discounts, Sarepta expects the net price to be about 20% lower, Chief Executive Officer Doug Ingram said on a call with analysts Thursday. Sarepta will charge $3.2 million for the one-time treatment, branded Elevidys. While the decision strikes a middle ground after questions arose about the treatment’s effectiveness, parents of older children will likely oppose the limitation. 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. (Bloomberg) - 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. ![]()
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