Shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) plunged 10% after the company disclosed that the U.S. Food and Drug Administration (FDA) has imposed a clinical hold on several of its gene therapy programs targeting limb girdle muscular dystrophy (LGMD).
The hold impacts a series of Sarepta’s experimental therapies, including SRP-9003, SRP-9004, SRP-6004, and SRP-9005. Notably, Sarepta had already paused most of these trials in recent days amid a company-wide restructuring, although SRP-9003 had remained active.
In a further setback, the FDA rescinded its earlier platform technology designation for Sarepta’s AAVrh74 gene therapy platform — a designation that had only been granted on June 2, 2025. Despite the regulatory headwinds, the company expressed plans to work with the agency to explore a potential Biologics License Application for SRP-9003 once the hold is lifted.
The FDA’s decision comes on the heels of a recent report by Reuters revealing that Sarepta had been asked by regulators to halt shipments of its Elevidys gene therapy following the death of a muscular dystrophy patient enrolled in a separate experimental treatment. Sarepta partially complied, pausing shipments for non-ambulatory patients but continuing deliveries for ambulatory patients.
Following the news, multiple Wall Street analysts revised their outlooks on the biotech firm. Piper Sandler’s Biren Amin downgraded Sarepta’s price target to $18.00 from $32.00 but held a Neutral rating. Cantor Fitzgerald analyst Kristen Kluska echoed the Neutral stance, assigning a $14.00 price target.
TD Cowen analyst Ritu Baral, maintaining a Hold rating and $28.00 price target, said,
“Patterns of lack of disclosure undermine SRPT’s credibility with DMD patient groups, the power behind prior approvals. While pt groups were aware of the patient deaths, they are, and have been for some time, frustrated with SRPT’s lack of disclosure on the overall safety of Elevidys, including cases of sudden cardiac death post-Tx as well as rates of severe liver events/ALI. SRPT’s arguments this past week on the relevance of LGMD events have further eroded confidence in their disclosure ethos. We strongly believe that patient group support behind SRPT has greatly eroded since winter.”
The regulatory challenges and scrutiny around transparency have clouded Sarepta’s near-term outlook, placing pressure on its investor and patient relationships alike.
Sarepta Therapeutics stock price
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