Even before the spate of deaths linked to Sarepta’s gene therapies last year, families of patients with Duchenne muscular dystrophy who had received Elevidys were frustrated. Sarepta’s flagship product, Elevidys is the only approved gene therapy for the rare degenerative disease, but the one-time treatment costs $3.2 million and recent data drops have left the DMD community and investors wanting.
“You don’t think you can do a little more effort and give us a little bit more, a little bit more information?” Catherine Collins, a parent of then 18-year-old Dylan, criticized at a patient advocacy conference in 2024. “We’re going to turn on you. We’re the people who give you the millions of dollars, but you’re not giving us anything back, which is facts, data and science. You’re just taking money.”
Then came the year that was 2025, when two Elevidys-linked deaths in the spring started a downward spiral for Sarepta’s stock. Over the summer, news broke of a third death, linked to an investigational gene therapy for limb-girdle muscular dystrophy that uses the same AAV vector as Elevidys. All told, the company’s share price plummeted 80% from the beginning of the year, reaching its lowest point in nearly a decade.
If company executives had hoped a recent 2025 earnings call and new three-year data for Elevidys could right the ship, they will be disappointed. While William Blair called the data an “incremental positive for Sarepta,” but “we do not think the data will significantly impact Elevidys prescribing and uptake behavior in the near term.”
Still, Sarepta is fiercely defending the therapy. Louise Rodino-Klapac, president of research and development and technical operations at Sarepta, defended the gene therapy in an interview with BioSpace. “The data speaks for itself,” she said. “We’re seeing an over four-point difference on NSAA [North Star Ambulatory Assessment]. This effect has grown over time, and I think that’s what’s really important.”
But the stock has dropped another 12% since the announcement last week, and experts who spoke with BioSpace were unconvinced by Sarepta’s case.
After looking at the three-year data, Mitchell Kapoor, senior analyst at H.C. Wainwright, put it bluntly: “I don’t know if [Elevidys] works,” he told BioSpace in an interview.
Now, three years after Elevidys was approved as the first gene therapy for DMD, Sarepta is facing potential competitors. Closest to market is REGENXBIO, which revealed last month a plan to file for approval in mid-2026 for the gene therapy RGX-202. If such options do reach the market, DMD patients and their families may well look beyond Elevidys for treatment.
With sales falling and no clear answers for encroaching competition, Sarepta could have more tough times ahead. And with a frustrated patient base and a tarnished brand, the biotech wouldn’t even make an attractive buyout option for a Big Pharma, according to Courtney Rice, principal at Acadia Strategy Partners.
“It’s like buying asbestos, or talc,” she told BioSpace. “I don’t think they’re built to last. It’s a story that’s gone on too long. Their best days are behind them.”
Through the Floor
Sarepta shared preliminary fourth-quarter 2025 earnings last month, beating consensus estimates with $369.6 million in total sales for the quarter. However, Elevidys missed its quarterly sales goals, bringing in about $10 million less than the $120.5 million consensus. The company attributed that miss to a severe flu season at the end of the year and to rescheduling six patient infusions from the fourth quarter into 2026.
Sarepta also confirmed an expected full-year 2026 sales floor of $500 million for Elevidys. But according to Kapoor, there are signs that the company isn’t going to hit that in the year ahead, with Elevidys sales dropping precipitously over the course of 2025.
Calling that sales floor a “stress-test scenario,” Kapoor said Sarepta is currently “below that on a run-rate basis.” If the therapy’s sales don’t pick up, Elevidys will miss the target by a considerable margin.
That could further dent Sarepta’s reputation on Wall Street, Kapoor said. “The Street has always been really optimistic about Sarepta,” he noted, but the company’s commercial pipeline is “neither projected to grow or even hold steady. They really need something to freshen up the story.”
On the Jan. 12 earnings call, Sarepta committed to more aggressive sales efforts in an attempt to reverse the trend. This includes hiring more sales representatives, running a promotional campaign and “pursuing initiatives with the patient community with the intention of increasing thoughtful and accurate communication,” William Blair analysts wrote after the call.
Only time will tell if those efforts will work, especially with REGENXBIO nipping at Sarepta’s heels. During an earnings call in August 2024, Sarepta CEO Doug Ingram said that “with no near-term gene therapy competition” the company expected to keep treating DMD patients for the remainder of the decade. But last month, Ingram referred to “other programs that are exciting in the gene therapy world” for DMD, seemingly admitting that those other competitors have arrived.
“They backtracked that narrative” about having complete control of the DMD market, Kapoor said.
In response to some of the criticism aimed Sarepta’s way, Rodino-Klapac held firm. “For Sarepta . . . they get unfairly scrutinized when they are the pathfinders and the pioneers in this space.”
Nevertheless, DMD patients and their families may soon face difficult decisions, as Elevidys uses the same vector as up-and-coming therapies, REGENXBIO’s RGX-202 and Solid Biosciences’ SGT-003.
“You can only AAV once,” Rice explained, so “if you pick Sarepta’s treatment you’re potentially passing up alternatives.”
REGENXBIO intends to file a biologics license application for accelerated approval for RGX-202 this year, while Solid last year reported Phase 1/2 data showing that SGT-003 improved muscle health and resilience.
If such options do reach the market, Rice expects the DMD community to move away from Sarepta’s gene therapy. “There’s resentment there; parents are hungry for an alternative,” she told BioSpace. “They’ve been held captive by [Elevidys] being the only thing on the market.”
https://www.biospace.com/drug-development/sarepta-saga-has-gone-on-too-long-as-competitors-catch-up
