Sarepta Therapeutics’ chief scientist suggested that the FDA was ahead of schedule in reviewing the company’s application to expand the label of its Duchenne muscular dystrophy gene therapy Elevidys.
Louise Rodino-Klapac“We’ve been informed by the agency that they will be sending us the draft labels sooner than they had previously indicated,” Sarepta’s top scientist Louise Rodino-Klapac said during an earnings call Wednesday afternoon.
“We should be receiving a draft of the Elevidys label very shortly,” CEO Doug Ingram echoed later in the call.
The FDA has a deadline of June 21 to make two calls on Sarepta’s gene therapy: whether or not to expand its label, and whether to convert it from an accelerated into a full approval. Elevidys received a narrow accelerated approval last year for boys ages 4 and 5 with the degenerative muscle condition.
The agency’s decisions around the Duchenne gene therapy have been closely watched by patient groups as well as by gene therapy developers, who view it as an indicator of the FDA’s evolving flexibility around regulating genetic medicines for rare diseases. Peter Marks, FDA’s top official on cell and gene therapies, has repeatedly spoken highly in favor of using accelerated approvals for more rare disease gene therapies.
Sarepta shared key results from a confirmatory clinical trial in October, reporting that it failed to meet its primary endpoint. But Sarepta pointed to secondary endpoints as a signal of efficacy, and the company is aiming for a broad label without restrictions on age or ability to walk.
Wall Street analysts have previously suggested that US regulators might make their decision earlier than expected.
Elevidys generated $133.9 million in revenue in the first quarter of the year. Sarepta did not take questions on its earnings call, citing a quiet period since Elevidys is undergoing FDA review.