FDA slaps a partial hold on Avidity’s mAb-siRNA therapy, pausing enrollment in muscle weakness disease study

Avidity Biosciences will have to wait to enroll new patients in a Phase I/II study of its therapy for myotonic dystrophy type 1, or DM1, which weakens muscles and leads to respiratory and cardiac issues.

The FDA is docking new enrollment in the MARINA study as the company and one of its trial investigators look into a serious adverse event reported in a patient in the 4 mg/kg cohort, the biotech said Tuesday morning. Patients currently enrolled can continue on the study drug or placebo and roll over into the open-label extension.

On an investor call, executives repeatedly declined to describe the adverse event, when it occurred or the status of the patient, citing privacy concerns. Asked if the patient was still alive, the company said yes.

Cowen analysts wrote in a note that the patient was in the hospital for tests and has since been discharged home. “While occurrence…
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