Sarepta Therapeutics Inc. shares fell the most in a month after the US Food and Drug Administration delayed its decision on the company’s gene therapy for Duchenne muscular dystrophy and said it was leaning toward clearing it for only for a subset of younger kids with the disease.
(Bloomberg) — Sarepta Therapeutics Inc. shares fell the most in a month after the US Food and Drug Administration delayed its decision on the company’s gene therapy for Duchenne muscular dystrophy and said it was leaning toward clearing it for only for a subset of younger kids with the disease.
The FDA told Sarepta it needs more time to complete its review, including to negotiate the label for the drug and post-marketing commitments, according to a statement from the company Wednesday. The agency is now expected to rule on the application by June 22; it had been scheduled to make its decision by May 29. The shares fell as much as 10% at the New York market open, the most intraday since April 13.
Sarepta said the agency has also indicated it is likely to grant accelerated approval only for Duchenne patients between the ages of 4 and 5. That age group seemed to benefit in a clinical trial that compared the therapy to a placebo, whereas there was no sign that it slowed the progression of disease in older children.
“The potential limitation of the population to 4-5 year olds is incrementally negative, as this would restrict the initial eligible population,” RBC Capital Markets analyst Brian Abrahams said in a note to clients. Overall, he called the news “a mixed bag,” as it still appears likely the drug will be approved, even if the age range might be limited at first.
Duchenne muscular dystrophy is a genetic condition that primarily affects boys, causing muscles to weaken over time. Most people with the condition die by their early 20s, making people eager for new treatments.
If the drug received accelerated approval, the company would have to confirm the benefit in a larger,ongoing placebo-controlled trial that’s expected to be completed later this year. It would be the first gene therapy for Duchenne muscular dystrophy.
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