After several delays and a narrow advisory committee vote, Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy has finally won an accelerated approval.
The FDA’s approval for Elevidys comes one month behind the agency’s prior schedule and in a restricted patient population. But a win is a win for Sarepta.
Specifically, the drug is approved to treat ambulatory patients ages 4 to 5 years with a confirmed mutation in the DMD gene.
Sarepta is charging Elevidys, a one-time gene therapy, at a list price of $3.2 million, CEO Douglas Ingram told investors during a call Thursday. He pointed to an analysis suggesting the drug could be cost-effective at $5 million.
Ingram expects the launch to take a few months to pick up, based on logistical and policy issues to address, he told investors.
As opposed to an treatment outcome-based payment approach that has been adopted by other existing gene therapies to manage cost, Sarepta is working on “some innovative concepts” with payers, the chief executive said.
Elevidys delivers a micro-dystrophin-encoding gene into a patient’s muscle tissue to prompt production of the micro-dystophin protein, which is necessary for patients with the disease because they can’t make the protein properly on their own.
The lack of this protein leads to a progressive loss of muscle strength. Sarepta’s treatment is meant to change the disease trajectory to a milder form.
Despite some hesitations about the drug within the FDA, the agency followed its advisory committee’s narrow 8-6 vote in favor of the therapy’s risk-benefit profile. The treatment’s clinical safety data, which included 13 serious side effects in 85 patients but no deaths or discontinuations due to adverse events, played a big part in the committee’s call during a meeting last month.
The vote came after the FDA flagged concerns in briefing documents ahead of the committee meeting. In its review, the agency said that the “the clinical studies conducted to date do not provide unambiguous evidence” that the treatment is “likely beneficial” for the patient pool.
It hasn’t been an easy road for the gene therapy. Over its six-year development period, it’s been put on two clinical holds, one in 2018 and another in 2021, both as a result of safety concerns.
After the recent advisory committee vote, the FDA pushed back its decision date on the drug by one month. At the time, Sarepta noted that the agency indicated it was “working toward potentially granting an accelerated approval” for the drug in the restricted patient population.
Sarepta also studied the drug in patients ages 6 and 7, but the data was murkier than in the younger age group. It hopes to clear up Elevidys’ effectiveness with a confirmatory trial that will be used for a potential label expansion to take off the age restrictions. The FDA has assured the company that it will consider that data at “maximum speed,” Ingram said in the call.
The last patient visit in that study is slated for September and Sarepta hopes for a readout shortly after with a potential expansion during the first half of next year.
As for the confirmatory study in relation to coverage, Ingram said the company has “never had a hint” from payers that reimbursement will have to wait on the upcoming trial results. The company has had “very good conversations” that allow it to be “very confident” about reimbursement, he said.
Editor's note: The story has been updated with Elevidys' price and additional details.