FDA grants orphan drug designation to Agios’s mitapivat for thalassemia

Agios Pharmaceuticals (NASDAQ:AGIO) +4.4% AH as the company receives FDA orphan drug designation to its pyruvate kinase-R (“PKR”) activator mitapivat for thalassemia.

Mitapivat was previously granted orphan drug designation by the FDA and EMA for pyruvate kinase (“PK”) deficiency, a rare, debilitating, hemolytic anemia.

https://seekingalpha.com/news/3581331-fda-grants-orphan-drug-designation-to-agioss-mitapivat-for-thalassemia