After an unconventional public display of disapproval, the FDA has made a stunning reversal on Sarepta Therapeutics’ Elevidys, recommending that Duchenne muscular dystrophy (DMD) patients who can walk can start taking the gene therapy again.
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days after the agency requested that the company halt all shipments of the one-time treatment following reports of three deaths among patients who had received a Sarepta gene therapy, including two on Elevidys.
In its own statement Monday, Sarepta said shipments to sites of care will resume imminently.
“Shipments of Elevidys remain halted for non-ambulant patients, which is the patient population where the treatment-related deaths occurred,” a Sarepta spokesperson confirmed to Fierce Pharma. “We continue to work with FDA on risk-mitigation measures for that patient population, which include discussions around enhanced immunosuppression.”
The FDA’s sudden change in course is a head-scratcher from a scientific perspective. In its July 28 release, the agency said it had concluded that the death of an 8-year-old in Brazil was not related to Elevidys but addressed none of the three prior deaths that triggered the pause. The three deaths, including one 51-year-old who got an investigational Sarepta product to treat another form of muscular dystrophy, were all caused by acute liver failure in non-ambulatory patients.
“The FDA will continue to work with the sponsor regarding non ambulatory patients, which remains subject to a voluntary hold, following two deaths,” the agency said.
The FDA requested the total shipment suspension—which was rare for a drug with a full FDA approval—and launched an investigation into the liver-toxicity-related deaths on July 18, when the death of the 8-year-old was not yet known to the wider public. Sarepta initially rejected the FDA’s request but soon reversed course.
Sarepta said it logged the 8-year-old’s case into the FDA’s adverse event report system in June. But the incident was not separately communicated to the public until last week.
As Sarepta temporarily stopped Elevidys supplies, word surfaced that the FDA was eyeing new study requirements to validate the drug’s safety before backing its use again in DMD patients. But that does not seem to be the case now.
Prior to the latest safety concerns, patient advocates had played a key role in the FDA’s original approvals of Elevidys despite the drug’s controversial clinical data. Now, opposition from the DMD patient community appears to have played a role in the FDA’s U-turn.
“The patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD,” the FDA said in its July 28 release.
“The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta,” the company’s CEO Doug Ingram said in a statement.