Home / Health / FDA Halts Rare Muscle Disease Trial Mid-Stage
FDA Halts Rare Muscle Disease Trial Mid-Stage
5 Mar
Summary
- FDA placed a partial clinical hold on PepGen's rare muscle disease drug trial.
- The hold stems from earlier laboratory and animal study concerns.
- Trial continues in South Korea, Australia, and New Zealand despite the hold.
The U.S. Food and Drug Administration has placed a partial clinical hold on PepGen's mid-stage trial for its drug targeting myotonic dystrophy type 1, a rare genetic muscle disease. This development caused PepGen's stock to plummet by more than 25% in after-hours trading on Wednesday, March 4, 2026.
The FDA's concerns are reportedly linked to earlier laboratory and animal studies submitted by PepGen. Importantly, the regulator did not identify any safety issues based on patient data from an initial-stage study. PepGen is actively collaborating with the FDA to resolve these questions, planning to submit additional information.
While the trial is on hold in the United States, PepGen has received clearance to continue the study in South Korea, Australia, and New Zealand. Patients in the UK and Canada are already participating and receiving the 10 mg/kg dose, following a favorable recommendation from an independent safety board regarding dose escalation. No patients had been enrolled in the U.S. portion of the trial prior to the hold.
