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The gene therapy will resume shipments for patients who are ambulatory.
Sarepta is resuming shipments of Elvidys for ambulatory patients.
Stock.adobe.com
Sarepta’s stock price may be recovering.
Share prices appear to be reversing course after they dropped significantly earlier this month after FDA raised new concerns about the safety of the company’s gene therapy Elevidys. As Pharmaceutical Executive previously reported, FDA asked the company to halt shipments of the drug and place all clinical trials on hold while it investigated three deaths of patients taking the drug. After rejecting these calls, Sarepta announced on July 21 it would voluntarily pause shipments of the drug within the US.
At the time, Sarepta issued multiple statements stating that no new safety signals had emerged amongst patients taking Elevidys. The company’s stock dropped about 36% in one day, however, and analysts downgraded the company.
On Friday, July 25, FDA issued a statement that it was investigating the death of an 8-year-old boy in Brazil who had been taking Elevidys.1 According to the agency’s statement, the death occurred on June 7 of this year. Later that same day, however, Sarepta fired back with its own statement that clarified that the boy’s death had been ruled unrelated to the treatment.2 The company also stated that it had reported the incident to FDA on June 18.
On July 28, Sarepta issued a statement confirming that FDA had removed its recommendation to halt shipments of the gene therapy for patients who are ambulatory.3 The statement also confirmed that FDA’s investigation into the Brazilian death came to the same conclusion as local health authorities: Elevidys was not responsible.
In the statement, Sarepta’s chief executive officer Doug Ingram said, “Last week, at the suggestion of FDA, Sarepta made the difficult decision to pause shipments of ELEVIDYS to provide the FDA with an opportunity to complete a review of available safety information. We are very pleased that FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of ELEVIDYS for ambulatory patients. The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta. 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 non-ambulatory patients, who remain on pause pending the outcome of those discussions.”
While MSN reports that Sarepta’s stock price began to rise on Monday on the heels of the announcement,5 the company is still facing challenges. Shipments of the gene therapy for non-ambulatory patients are still halted, and other incidents are still being investigated.
Sarepta is also facing a class action lawsuit that claims investors were adversely affected by securities fraud between June, 2023, and June, 2025.4 The suit alleges that the company made false statements about the safety risks related to the gene therapy. Law firm Levi & Korsinsky is representing the plaintiffs and is actively seeking investors who wish be named as plaintiffs in the case.
However, the recent announcement has alleviated some investor concerns. In mid-July, Sarepta’s stock hit a monthly-high of $21.97-per-share. After dropping close to $10-a-share, it has since climbed up to about $16-a-share as of Tuesday afternoon. The stock has been steadily declining over the year, but the latest news is still positive for the company. Analysts say that concerns over the company’s ability to stay in business have been reduced.
For a full timeline of Sarepta’s recent struggles, follow this link for Pharmaceutical Executive’s coverage.
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