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Investing.com -- Sarepta Therapeutics Inc (NASDAQ:SRPT) stock tumbled 10% following the announcement that the U.S. Food and Drug Administration has placed a clinical hold on the company’s investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD).
The FDA’s decision affects multiple Sarepta LGMD programs, including SRP-9003, SRP-9004, SRP-6004, and SRP-9005. This comes just days after the company had already paused most of these programs as part of a strategic restructuring process, with the exception of SRP-9003.
Additionally, the FDA has revoked the platform technology designation for Sarepta’s AAVrh74 platform technology that was previously granted on June 2, 2025. The company stated it intends to discuss with the FDA a potential pathway to submit a Biologics License Application seeking accelerated approval for SRP-9003 after the clinical hold is lifted.
The clinical hold follows recent controversy reported by Reuters, where U.S. regulators asked Sarepta to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died. Sarepta declined to fully comply, continuing shipments to ambulatory patients while maintaining a halt for non-ambulatory patients implemented on June 15.
Several analysts adjusted their outlook on Sarepta following the news. Piper Sandler analyst Biren Amin lowered the price target to $18.00 from $32.00 while maintaining a Neutral rating. Cantor Fitzgerald analyst Kristen Kluska reiterated a Neutral rating with a $14.00 price target.
TD Cowen analyst Ritu Baral, who maintained a Hold rating and $28.00 price target, commented, "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."
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