After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
Sarepta Therapeutics will comply with an FDA request to temporarily stop all U.S. shipments of its Duchenne muscular dystrophy therapy Elevidys, walking back its previous decision to keep the gene therapy available to patients despite mounting safety concerns and regulatory scrutiny.
In an announcement on Monday evening, the Massachusetts biotech said that the move will give it enough time to respond to the FDA’s information requests regarding Elevidys, and allow the regulator and the company to complete the supplemental safety labeling process. Shipments will be suspended starting at the close of business Tuesday, July 22.
Writing to investors soon after Sarepta’s announcement, analysts at Jefferies said that the decision to stop shipments “makes sense to us, since a major hospital . . . has just paused dosing anyway,” referring to the Children’s Hospital of LA. Still, with Elevidys paused, the drug’s sales outlook “now faces an additional uncertainty,” the analysts wrote.
“The key questions are how long the pause will remain in effect, and how much of Elevidys’ adoption in ambulatory DMD [Duchenne muscular dystrophy] will ultimately be affected,” Jefferies continued. The suspension, the analysts emphasized, is voluntary in nature, which means that Sarepta “can technically elect to resume Elevidys shipments at any time.”
Sarepta has reported three patient deaths in recent months, the first two of which are linked to Elevidys. News of the first mortality came in March, the second in June and the third last week—all of which were attributed to acute liver failure, a known complication of adeno-associated virus vector-based gene therapies. The last of the reported deaths were reported in a patient treated with SRP-9004, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD). Sarepta stopped shipping Elevidys for non-ambulatory patients on June 15.
These three patient deaths have made Sarepta’s story “challenging to navigate,” Jefferies said in its Monday note. “Looking ahead, we still expect more patients/families/physicians to rethink therapy, and wouldn’t be surprised if the patient community grew more divided.”
In response to the announcements of these deaths, the FDA on Friday requested that Sarepta suspend all of its Elevidys shipments in the U.S., including in ambulatory patients, but the company initially refused to comply. “We will continue to ship Elevidys to the ambulant population,” Sarepta said on its own news release on Friday, noting that its “comprehensive scientific interpretation of the data” shows “no new or changed safety signals” in these patients.
Sarepta has now walked back on that initial refusal, citing the need to stay on good terms with the FDA. “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension,” Sarepta CEO Doug Ingram said in a prepared statement alongside Monday’s press announcement.
The FDA on Friday also suspended all of Sarepta’s clinical trials of LGMD and took back its Platform Technology Designation for the company’s vector platform, which it uses to deliver its gene therapies.
