FDA grants breakthrough status for myotonic dystrophy type 1 RNA therapy

Today, the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to delpacibart etedesiran (AOC 1001).

This is USA-based Avidity Biosciences’ (Nasdaq: RNA) lead clinical development program for the treatment of myotonic dystrophy type 1 (DM1), with the news pushing the firm’s share up more than 2% to $25.90.

DM1 is a rare, underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Delpacibart etedesiran (or del-desiran), based on the company’s proprietary AOC platform, consists of a monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting DMPK mRNA, the underlying cause of DM1.