Ionis' (IONS) Olezarsen Gets FDA's Orphan Tag for Rare Disease

Ionis (IONS) recently announced that the FDA has granted orphan drug designation to its pipeline candidate, olezarsen, for the treatment of familial chylomicronemia syndrome (FCS), a rare genetic disease.The FDA’s orphan drug designation is bestowed on medicines in development for the treatment, prevention or diagnosis of a rare disease that affects fewer than 200,000 people in the United States at the time of designation. The rare disease, FCS, is characterized by extremely high triglyceride levels and rec ...