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.
(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.
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.
Sarepta will charge $3.2 million for the one-time treatment, branded Elevidys. After discounts, Sarepta expects the net price to be about 20% lower, Chief Executive Officer Doug Ingram said on a call with analysts Thursday.
Shares of Sarepta were little changed in extended trading in New York.
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. The inherited condition primarily affects boys, causing their muscles to weaken over time. Most people with the condition die by their early 20s, making people eager for new treatments.
The company plans to seek permission to treat older patients depending on the results of a forthcoming trial, Ingram said.
The urgent need for a treatment helped Sarepta win over independent advisers to the FDA at a recent hearing on the drug. They recommended the therapy be approved in an 8-to-6 vote, with some panelists explaining their desire to give an option to patients and their families who pleaded for the treatment to be approved.
People with Duchenne muscular dystrophy, or DMD, don’t make enough dystrophin, a protein that supports muscles. Sarepta’s one-time gene therapy helps people make a much smaller version of that protein, called micro-dystrophin. However, some panelists and FDA reviewers have expressed skepticism that the two proteins are interchangeable and that the drug will provide much benefit.
Sarepta will need to confirm its therapy produces clinical benefit in a larger trial in order to receive a full approval and keep the drug on the market. The company has said that study is fully enrolled and should be completed later this year.
If those results are positive, Sarepta anticipates it could expand use of Elevidys to more patients in the first half of next year, Ingram said.
(Updates with price, CEO commentary in third, fifth and final paragraphs.)
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