Agios Pharmaceuticals files NDA for drug that would treat pyruvate kinase deficiency

The small-molecule therapeutics company Agios Pharmaceuticals (NSDQ:AGIO) has submitted a new drug application (NDA) with the FDA to use mitapivat to treat adults with pyruvate kinase (PK) deficiency, a rare condition in which the rapid breakdown of red blood cells occurs can trigger anemia. The condition is the result of an inherited lack of the enzyme PK.

Mitapivat is a novel oral, small molecule allosteric activator of wild-type and various mutated PKR enzymes.

“With this NDA filing, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong hemolytic anemia characterized by serious complications affecting multiple organs,” said Dr. Chris Bowden, the chief medical officer at Agios.

There are currently no FDA-approved therapies for PK deficiency. While mild cases may not require treatment, physicians treat more severe forms of the disease with blood transfusions and splenectomy. PK defici…

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