Avidity’s del-desiran for DM1 named orphan drug in Japan

Avidity’s del-desiran for DM1 named orphan drug in Japan

The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare diseases affecting fewer than 50,000 patients in Japan. An investigational therapy is eligible if no approved treatment op…

Cellenkos Announces US FDA Orphan Drug Designation Granted to CK0801 (Allogeneic Cord Blood derived T regulatory Cell Product) for Treatment of Aplastic Anemia

Orphan Drug Designation granted for Aplastic AnemiaCK0801 is planned for Registration trial in H2 2025