FDA Approves Agios' Pyrukynd To Treat Hemolytic Anemia In Adults With Pyruvate Kinase Deficiency

(RTTNews) - Agios Pharmaceuticals Inc. (AGIO), said on Friday that the FDA has approved Pyrukynd for the treatment of hemolytic anemia in adults with pyruvate kinase or PK deficiency, a rare, debilitating, lifelong hemolytic anemia.

Pyrukynd, generically known as Mitapivat, is a first-in-class, oral PK activator and the first approved disease-modifying therapy for this disease, the company said.

Pyrukynd is expected to be available in the U.S. approximately two weeks after approval. The drug was reviewed by the FDA under priority review and was previously granted orphan drug designation.

The product is also under review by the European Medicines Agency (EMA) as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU by the end of 2022.

PK deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.