Avidity biosciences receives orphan drug designation in japan for delpacibart etedesiran (del-desiran) for treatment of myotonic dystrophy type 1

Del-desiran first-ever investigational treatment for dm1 to receive orphan drug designation in japan  san diego , april 8, 2025 /prnewswire/ -- avidity biosciences, inc. (nasdaq: rna), a biopharmaceutical company committed to delivering a new class of rna therapeutics called antibody oligonucleotide conjugates (aocs™), today announced that the japan ministry of health, labour and welfare (mhlw) has granted orphan drug designation (odd) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (dm1), an investigational treatment designed to address the root cause of dm1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. del-desiran is the first investigational treatment for dm1 to receive orphan drug designation in japan.
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