FDA Orphan Drug Designation granted to mitapivat for treatment of thalassemia

Mitapivat, an oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R enzymes, was previously granted orphan drug designation by FDA and European Medicines Agency for pyruvate kinase deficiency, a rare, debilitating, haemolytic anaemia.


Biospace Inc.