The Food and Drug Administration on Thursday approved Sarepta Therapeutics’ one-time gene therapy treatment for children with Duchenne muscular dystrophy, despite unanswered questions about the drug’s clinical benefit.
The decision marks the first approval of a gene therapy to treat DMD, a fatal and progressive disease that manifests in young children, causing muscle atrophy that leads to disability and death.
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The FDA’s approval of the gene therapy marks a win for Sarepta.
Dreamstime
The Food and Drug Administration on Thursday approved Sarepta Therapeutics ’ one-time gene therapy treatment for children with Duchenne muscular dystrophy, despite unanswered questions about the drug’s clinical benefit.
The decision marks the first approval of a gene therapy to treat DMD, a fatal and progressive disease that manifests in young children, causing muscle atrophy that leads to disability and death.
It’s also one of the few times the FDA has granted accelerated approval to any gene therapy; accelerated approval allows a drug to be sold based on laboratory measures thought to predict clinical benefit, rather than direct evidence of that benefit.
The FDA limited its approval of the Sarepta (ticker: SRPT) drug—which the company will market under the name Elevidys—to children ages 4 and 5 who are still able to walk.
Sarepta had tested the drug in children ages 4 to 7 years old, and had asked the FDA for approval in all DMD patients still able to walk, which could include some individuals as old as 13.
While the approval is narrower than Sarepta had hoped, it still marks a win for the company, particularly since FDA staff had initially intended to reject the application for the drug’s approval entirely, according to reports from earlier this year.
In mid-May, a panel of outside FDA advisors narrowly voted to recommend approval based on data showing the drug causes DMD patients to produce a version of a protein that is linked to muscle growth, and which their bodies cannot otherwise produce.
Sarepta hasn’t yet produced clear evidence that the gene therapy can stop muscle decline in patients. Its aggressive decision to seek approval for the drug before the completion of a continuing Phase 3 trial shocked Wall Street last year. That Phase 3 trial is expected to produce top-line results late this year.
In a statement, the FDA said it would examine the results of that trial once they were available.
“A clinical benefit of Elevidys, including improved motor function, has not been established,” the FDA said. “The agency will review the data from this trial as quickly as possible to consider if further action, such as a revised indication or withdrawal of Elevidys, may be necessary.”
Still, at least for now, the company’s bet seems to have paid off. Sarepta shares have climbed 77% over the past 12 months. In late May, Sarepta shares fell after the FDA delayed its decision date on the drug. At the time, Sarepta signaled that if the FDA did approve the gene therapy, it would likely do so for a narrower age cohort than the company had hoped.
Shares of Sarepta were roughly flat Thursday afternoon.
The company didn’t say in its initial news release what it intends to charge for the gene therapy. Sarepta is holding an investor call to discuss the approval at 4:30 p.m. Eastern Thursday.
“This approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” said Sarepta CEO Doug Ingram in a statement.
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com