A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
About 90% of doctors surveyed by BMO Capital Markets are still prescribing Sarepta’s Elevidys, the Duchenne muscular dystrophy gene therapy that become subject to regulatory scrutiny earlier this summer after a pair of deaths.
The 30 doctors surveyed are keeping their scripts to just ambulatory patients, however, since the deaths occurred in patients who were non-ambulatory. More than a quarter of the physician respondents in BMO’s survey said they would prescribe Elevidys to younger and healthier patients.
The doctors cited the favorable risk/benefit ratio in ambulatory patients, more than 760 of whom have been treated, with zero deaths. These patients are generally healthier and lighter—meaning they only need the lower dose of Elevidys, as compared to non-ambulatory patients who tend to be older, more advanced in their disease and heavier, thus requiring the higher dose.
Elevidys is approved for ambulatory patients at least four years old who have a confirmed mutation in the DMD gene. It can also be used for non-ambulatory patients with a confirmed mutation. This latter indication was granted under the accelerated approval pathway, meaning Sarepta is required to confirm the benefit to be granted a full traditional approval.
The first death associated with Elevidys was reported in March, when Sarepta revealed that a boy who was unable to walk had died from liver injury after receiving the gene therapy. This is a known side effect of gene therapies that use AAV vectors for delivery. A second death occurred in June in a similar patient, resulting in a black box warning on the Elevidys package warning of the potential risks for non-ambulatory patients.
Then in July, a third death occurred in a clinical trial for another of Sarepta’s gene therapies for limb-girdle muscular dystrophy that also used an AAV vector. While not the same as Elevidys, the death raised concerns about the future of AAV gene therapies.
The FDA then stepped in and asked Sarepta to halt shipments of Elevidys. After some drama between the company and the regulator, the brief hold was lifted at the end of July.
But the damage to Sarepta had been done. BMO notes that as of Tuesday, the company is trading down 85% year to date. The stock opened at $17.47 Tuesday morning, compared to $124.09 on the first trading day of 2025.
Nevertheless, there is still demand for Elevidys, although it’s slightly muted. BMO reports that there were some cancelled appointments amid the FDA drama but 70% of the surveyed physicians said they are seeing similar to slightly lower demand for Elevidys now as compared to the period around when the deaths were reported.
Since Elevidys returned to the market on July 28, doctors said they had collectively infused 27 patients and 12 more are scheduled between the third quarter 2025 and first quarter 2026. This reflects about $95 million in sales for the drug, which costs $3.2 million for the one-time infusion. When reporting second quarter earnings in early August, Sarepta beat estimates but recorded a 25% decline in sales for the gene therapy.
Part of Sarepta’s new suggested dosing guidelines include the use of the immunosuppressant sirolimus along with Elevidys to tamp down the elevated liver enzymes believed to have caused the deaths. According to BMO’s survey, 80% of the doctors are happy that this proposed change will improve the safety of Elevidys.
Should there be a death in the ambulatory patient population, however, half of the doctors said they would stop prescribing Elevidys and await further safety updates. That has so far not occurred in the market environment.
