Sarepta Therapeutics is under renewed scrutiny following the third patient death linked to its gene therapy programs, sparking analyst concerns over transparency and safety. The company disclosed that a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 died from liver failure after receiving investigational gene therapy SRP-9004 as part of a phase 1 trial.
Unlike two earlier fatalities tied to Elevidys, Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy, this incident involved a different experimental therapy. However, all three deaths involved liver toxicity, raising broader safety questions about the company’s gene therapy pipeline. Sarepta stated it had notified regulators and trial investigators about the latest incident and acknowledged the challenges of developing gene therapies for ultra-rare conditions.
The death adds to mounting criticism of Sarepta’s communication practices, particularly after it failed to disclose the fatality during its recent investor call and strategic restructuring announcement. That update included removing several LGMD programs—including SRP-9004—from its pipeline. CEO Douglas Ingram later justified the omission, saying the death was not material to the business update.
Analysts from William Blair and Leerink Partners have flagged the incident as a red flag for both current and future gene therapy programs. While Sarepta insists that discontinuing SRP-9004 was unrelated to the fatality, analysts believe the tragedy may have played a role in the decision. The scrutiny now extends to SRP-9003, another LGMD gene therapy aiming for FDA approval in late 2025. Given the shared viral vector with Elevidys, experts warn that regulators may heighten safety requirements and that commercial prospects could be limited even if approved.
Meanwhile, FDA Commissioner Martin Makary has signaled increased oversight of Elevidys, potentially revisiting its market approval status in light of these safety concerns.
Sarepta’s stock plummeted over 33% amid the controversy, driven by the delayed disclosure and deepening investor concern. In response, the company reaffirmed its commitment to transparency and confirmed there are no unreported serious safety events. Additionally, Sarepta is shifting focus to siRNA-based therapies, exploring potential partnerships or consolidations for its remaining gene therapy assets. The company also reported a fresh round of layoffs and acknowledged a black box warning for liver toxicity on Elevidys.
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