After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.
Weakening sales of Sarepta Therapeutics’ gene therapy Elevidys are not just a blip on the radar but a signal that the company will likely fall below a $500 million per year sales floor that was projected in January.
Duchenne muscular dystrophy gene therapy Elevidys brought in $110 million for the fourth quarter, finishing off the year with $898.7 million in total net sales. The dip was brought on by a severe flu season and six planned infusions that have been moved into 2026, CFO Patrick Moss explained on a fourth quarter earnings call on Wednesday.
But Sarepta also suffered after a rocky 2025 that saw multiple deaths linked to a viral vector that delivers Elevidys’ and an investigational gene therapy, Moss conceded. “The safety events of 2025 reshaped the perceptions of gene therapy and of Elevidys specifically.”
William Blair analysts said that revenues now seem likely to go well below the $500 million sales floor the executives reiterated in January.
“The Elevidys weakness in the fourth quarter is not an outlier but was an early signal of a materially weaker Elevidys trajectory in 2026,” the firm wrote on Thursday morning.
Sarepta now predicts that sales of Elevidys will be flat or down as far as 15%. CEO Doug Ingram, who on the call announced his intention to retire due to family obligations, said that three of the patients who had been delayed have already received their infusions. And a few patients can make a big difference with Elevidys, which is one of the most expensive drugs in the world at $3.2 million for a single dose.
“With Elevidys, the quarter-to-quarter dynamics, they can be noisy,” Moss said. “We have to consistently look at performance over the longer arc rather than each quarter.”
Sarepta could also soon lose marketing authorization for its other DMD products, the exon skippers Vyondys 53 and Amondys 45, after the failure of a confirmatory trial for the treatments, Willima Blair noted. Therefore, the firm views Sarepta’s “near-term upside potential as limited.”
Overall, Sarepta achieved $1.86 billion in revenue for 2025.
Time To Reset
Moss expressed confidence that Sarepta can rebound from the annus horribilis of 2025. To recap, Sarepta reported the deaths of two patients who had received Elevidys in March and June 2025. News of a third patient death—in this case a participant in a clinical trial for an investigational gene therapy for limb-girdle muscular dystrophy that uses the same gene therapy backbone as Elevidys—followed in July.
A very public battle with the FDA ensued, resulting in a more than week-long pause on Elevidys shipments and ultimately a label change to include only ambulatory patients. Sarepta voluntarily added a black box warning of serious liver toxicities and risk of death.
Sarepta announced a strategic pivot from gene therapy in July, with a focus on its partnership with Arrowhead Therapeutics.
“It has become more clear to us that 2026 serves as a critical reset year for the ambulatory patient population, where there’s an information imbalance and the disease-modifying benefits of Elevidys is less understood,” Moss said.
Sarepta unveiled long-term data for Elevidys in January showing the gene therapy’s ability to stabilize symptoms of the neuromuscular disorder three years after dosing. While some experts found these data underwhelming, Moss said the results should help the company rebuild the case for the gene therapy.
Change Ahead
In addition to its business woes, Sarepta will also now be forced to find a new leader. Ingram explained on Wednesday’s call that two of his immediate family members had been diagnosed with myotonic dystrophy (DM1), not long after Sarepta struck up the collaboration with Arrowhead, which included an investigational treatment for the devastating disease.
“Well, subsequent to that partnership in a fairly shocking and certainly ironic twist of fate, my personal commitment to muscular dystrophy is deepened,” he said.
Ingram has long been one of biopharma’s more polarizing figures. Sarepta’s regulatory approvals have been hard fought based on mixed trial data that often resulted in controversial approval processes. The events of 2025 had Ingram once again defending his company despite criticism from all sides.
“We set audacious goals for ourselves, chief among them, the goal of lessening the burden and extending the lives of boys and young men with [DMD],” Ingram said in his opening remarks. “Along the way, as you all know, we solved many thorny scientific and technical problems, even as we faced and overcame many exogenous and extraordinary obstacles—challenges that would have undone a less committed, less creative, less resilient organization.”
A search is now underway for Ingram’s successor. The CEO, who plans to exit by the end of the year, said the board is open-minded on an internal or external candidate but most important is a person who understands the battle-tested company’s culture.
“While investors have polarized views of CEO Doug Ingram, uncertainty about his successor could cause additional stock volatility in the near term,” William Blair wrote.
Still, Sarepta has some key events to look forward to that could help the biotech’s comeback, according to analysts from Jefferies. Data from that Arrowhead-partnered DM1 asset as well as from Sarepta’s FSHD candidate are due in the first quarter, Jefferies noted, adding that together the drugs could represent a $1 billion opportunity for Sarept . In addition, non-ambulatory data for Elevidys and sirolimus, which has been added to the regimen to mitigate the liver risk, are due in the second half.
Sarepta will meet with the FDA this quarter to discuss the future of its exon skippers, with a decision possible in the second quarter. Jefferies sees a full withdrawal as unlikely. The best-case scenario would be converting the accelerated approval to a traditional approval.
And Elevidys still has a chance to return to prominence in the second half of the year after a “trough” in the first six months, Jefferies said.
“We think a re-acceleration in H2:26 is possible, with management making a marketing/education push to highlight Elevidys’ positive benefit/risk profile in ambulatory DMD,” the Jefferies team wrote Wednesday evening.
