Biotech stories typically tread a very fine line between promise and pressure.
Sometimes a company that’s been a pioneer in rare diseases suddenly finds itself at a crossroads.
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With years of headline-making science behind it, one major biotech stock is now facing a moment that flips the script on how investors and regulators see its future.
It’s not about a singular decision, but a pattern coming into focus. What’s surfacing now is a test of just how far early bets can stretch.
Sarepta’s DMD journey shows promise, pressure, and tough questions
Sarepta Therapeutics (SRPT) has been at the core of the conversation around Duchenne muscular dystrophy (DMD) treatments, but it hasn’t been without its controversies.
Back in 2016, Sarepta won FDA approval for Exondys 51, the first drug to target a specific DMD mutation.
It got the approval through the agency’s accelerated pathway, which fast-tracks promising treatments to reach patients based on early data.
However, that decision sparked debate.
The clinical trial included just 12 boys, and while dystrophin, a key muscle protein, saw a boost, its skeptics questioned whether that translated into real-world benefits.
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Europe’s regulators dismissed the drug in 2018, and U.S. insurers placed tight restrictions on coverage.
Nevertheless, the therapy carved out a strong niche, serving a small group of patients with exon 51 mutations.
Sarepta followed things up with Vyondys 53, another exon-skipping treatment, and saw a similar pattern emerge.
It followed an initial pushback, eventual FDA approval, and ongoing requests for stronger data from insurers and clinicians.
Even with these challenges, the DMD market remains strong.
The disease affects close to 13,000 boys in the U.S., and treatments topped $2 billion in North American sales last year. Globally, that market could potentially reach $6.5 billion by 2034.
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Sarepta’s biggest bet came with Elevidys, a gene therapy priced at $3.2 million per dose. The treatment was estimated to help roughly 90% of U.S. DMD patients.
That massive scale and price reignited debate around cost, access, and evidence.
The company’s path has been ambitious.
However, with investor confidence shaken by safety questions and shifting strategy, the road ahead seems mostly rocky.
Sarepta faces fresh crisis as third gene therapy death reported
Sarepta Therapeutics is feeling the heat after a third patient death linked to its gene therapy program came to light.
Following the development, its stock tanked roughly 35% on July 18, having lost 88% year-to-date.
The company confirmed that an early-stage trial patient being treated for limb-girdle muscular dystrophy died last month from acute liver failure.
That news adds to the scrutiny over the safety of Sarepta’s experimental treatments.
This follows two earlier deaths tied to Elevidys, a gene therapy for DMD developed with Roche (RHHBY).
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Those patients were said to be in advanced stages of the disease and had lost their ability to walk.
Sarepta says it acted quickly, notifying regulators and investigators “in an appropriate and timely manner.”
Nonetheless, the fallout is growing. The company has shut down a related clinical trial and paused the distribution of Elevidys for non-ambulatory patients.
It’s discussing safety protocols for high-risk groups with the FDA.
Adding to the duress, Sarepta announced a massive corporate shake-up this week.
It rolled out a sweeping restructuring plan, cutting 36% of its workforce (500 jobs).
It’s also looking to stop producing multiple drug programs to focus more tightly on siRNA and gene-silencing therapies, which potentially could be a lot more promising pipeline bets.
Management expects these cost-cutting measures to save around $400 million annually starting in 2026.
Given the combination of patient deaths, halted trials, and a shift away from some therapies, Sarepta may now be entering a major reset phase.
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