FDA lifts hold on Avidity’s delpacibart etedesiran trial

The US Food and Drug Administration (FDA) has removed the partial clinical hold on delpacibart etedesiran (del-desiran/AOC 1001), an investigational treatment designed to address the root cause of myotonic dystrophy type 1 (DM1), under development by US biotech Avidity Biosciences (Nasdaq: RNA).

Del-desiran is being evaluated in the ongoing Phase III HARBOR trial in patients with DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

In 2022, the FDA placed a partial clinical holdon new participant enrollment in the Phase I/II MARINA clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).