Orphan Designation

• FDA

FDA Approves Orphan Drug to Treat Aplastic Anemia

On February 21, 2013, the U.S. Food and Drug Administration (“FDA”) granted orphan drug status to an aplastic anemia treatment.…

• FDA

FDA Approves New Orphan Drug (Elelyso) to Treat Gaucher Disease

On May 1, 2012, the U.S. Food and Drug Administration (“FDA”) approved a new orphan drug to treat Gaucher disease.…