Avidity Biosciences (RNA) said Tuesday it has received orphan drug designation from Japan's Ministry of Health, Labor and Welfare for delpacibart etedesiran, or del-desiran, to potentially treat myotonic dystrophy type 1, a neuromuscular disease.
The investigational drug has also received breakthrough therapy, orphan drug and fast track designations by the US Food and Drug Administration, and orphan designation by the European Medicines Agency, Avidity said.
The company said it expects to complete enrollment in a phase 3 trial in the middle of this year and file marketing applications starting 2026 in the US, European Union and Japan.
Avidity shares were rising more than 2% in early trading.
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