Sarepta stock took another dive Monday after the biotech company refused the Food and Drug Administration's request to voluntarily stop all shipments of its gene therapy, Elevidys.
The FDA's request comes after two patients with Duchenne muscular dystrophy died following treatment with the approved drug. Last week, Sarepta Therapeutics confirmed another patient died after receiving one of its experimental gene therapies for limb-girdle muscular dystrophy.
Analysts say the FDA is reacting to the public outcry, rather than the science. Leerink Partners analyst Joseph Schwartz says these are "unprecedented times" as the "saga continues to get messier."
Sarepta says it informed the agency of the third patient's condition — life-threatening acute liver failure — on June 20. Then, the company notified the FDA of the patient's death on July 3.
The FDA knew of the patient's death for two weeks before asking Sarepta to stop all shipments of Elevidys, Leerink Partners analyst Joseph Schwartz said in a report. In the same move, on Friday, the agency put all remaining limb-girdle muscular dystrophy studies on hold and revoked the platform designation for Sarepta's viral vector. All of the company's gene therapies share this delivery system.
"We have concerns that the agency's recent actions reflect a shift toward decision-making influenced more by social factors than by science or regulatory precedent," he said in a report. "This makes it increasingly more difficult to anticipate how it will govern moving forward."
Sarepta stock toppled 5.4%, ending the regular session at 13.32.
No New Safety Issues With Elevidys
It's important to note that the two Elevidys patients who died were older, ages 15 and 16, and could no longer walk. That means they were sicker and weighed more, requiring a higher dose of the drug. The company also added a warning to the label for Elevidys, noting the potential for acute liver failure.
The FDA's request on Friday pertains to patients who can still walk, or ambulatory patients.
Sarepta says there haven't been any "new or changed safety signals in the ambulant patient population." The third patient who died was a 51-year-old man with limb-girdle muscular dystrophy. He was enrolled in a study of SRP-9004. Sarepta announced a restructuring last week and, as part of that, paused that particular study.
The company was planning to soon ask the FDA to approve another limb-girdle muscular dystrophy gene therapy, this one called SRP-9003. But that agency also put that one on hold on Friday. William Blair analyst Sami Corwin notes this will extend Sarepta's timeline for that gene therapy.
She sees the timing of the clinical hold as "a bit peculiar" given the FDA already knew about the death on July 3. This suggests "the FDA's announcement is in direct response to the public becoming aware of the death and residual outcry rather than new information obtained by the FDA," she said in a report.
Corwin kept her market perform rating on Sarepta stock. She said formally removing Elevidys from the market could take two years or longer. Though it's possible, it's the less likely scenario, she said.
Follow Allison Gatlin on X/Twitter at @AGatlin_IBD.
