The Food and Drug Administration has given Sarepta Therapeutics a green light to resume shipping its gene therapy Elevidys to some patients with Duchenne muscular dystrophy, a little over one week after demanding the company halt sales over safety concerns.
In a statement Monday evening, Sarepta said it would begin shipments to treatment sites “imminently.” The resumption applies only to Duchenne patients who can still walk, which typically describes individuals who are younger and whose disease hasn’t advanced as far.
For Duchenne patients who can no longer walk, shipments remain on a volunatary pause Sarepta instituted in June following the death from liver failure of a second teenager who had received Elevidys. Both teenagers were no longer able to walk when they died.
“We look forward to working collaboratively with the FDA to complete the safety label update for Elevidys and to discussing the approach to risk mitigation for nonambulatory patients, who remain on pause pending the outcome of those discussions,” Sarepta CEO Doug Ingram said.
Sarepta had initially refused to comply with the FDA’s July 18 request to stop Elevidys shipments in ambulatory patients, precipitating a three-day standoff with the agency before the company chose to back down last Monday.
The FDA’s request came on the heels of a furor over Sarepta’s failure to disclose the death of a patient with a different kind of muscular dystrophy who received one of the company’s experimental gene therapies in a clinical trial. That experimental treatment and Elevidys share a common delivery component, but differ in dose, manufacturing process and genetic payload.
More recently, an 8-year-old boy in Brazil who had received Elevidys died, but his death was ruled unrelated to the gene therapy. In a statement Monday, the FDA said it had investigated that case and agreed.
“The FDA will continue to work with the sponsor regarding non ambulatory patients, which remains subject to a voluntary hold, following two deaths,” said the agency.
The FDA’s sudden change in stance caught multiple Wall Street analysts by surprise. Citing anonymous agency officials, several published reports last week that suggested the company would need new safety data before the FDA would endorse Elevidys’ return to market. Vinay Prasad, the new head of the agency’s gene therapy office, had also been highly critical of Elevidys even before the deaths of multiple treatment recipients.
But the FDA, in its statement, indicated it listened to the “important voice” of the Duchenne patient community, an educated and mobilized group that has influenced past agency choices and, in certain cases, has funding ties to drugmakers like Sarepta. Prasad’s decisionmaking and political leanings have also been targeted in recent days by conservative commentators and in opinion pieces published in the Wall Street Journal, noted Baird analyst Brian Skorney.
The agency’s “unexpected” reversal is likely due to a “mix of patient advocacy pressure and a rebuke of FDA leadership by higher-ups” in the Trump administration, Skorney wrote to clients. “We wouldn't be surprised to see a resignation in the short term based on how poorly the decision to request the halting of ambulatory shipments has played out.”
Elevidys’ renewed availability could relieve financial pressure on Sarepta, which is counting on a minimum of $500 million in annual sales from the therapy to remain viable. The FDA’s decision makes it increasingly likely the company can cover its debts, wrote William Blair analyst Sami Corwin.
Still, Corwin expects to see “some hesitancy” from patients and physicians once shipments resume. In a separate note, RBC Capital Markets’ Brian Abrahams expressed a similar view, predicting a decline in Elevidys use that would put yearly sales below the $500 million target.
Elevidys' stay on the market could be short-lived, too, as the company might have trouble figuring out which Duchenne patients might have an “acceptable” risk of potentially serious health problems, added Leerink Partners’ Joseph Schwartz.
“Even one death in an ambulatory patient could be the end of Elevidys,” Schwartz wrote on Monday.
Editor’s note: This story has been updated to include analyst commentary.
