FDA halts study of Sarepta treatment for Duchenne muscular dystrophy due to safety concern

Sarepta Therapeutics said Thursday that it has temporarily stopped a clinical trial of its second-generation medicine for patients with a certain type of Duchenne muscular dystrophy due to a serious safety incident reported by a patient.

The Food and Drug Administration placed a clinical hold on the Sarepta drug, called SRP-5051, after a patient in the study experienced a “serious” decrease in blood-based magnesium, a condition known as hypomagnesemia.

SRP-5051 belongs to a new class of treatments, which Sarepta refers to as its “PPMO platform,” that has become more important for the company following significant setbacks with its efforts to develop a one-time gene therapy for Duchenne.

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