Mitapivat from Agios scores FDA nod as for hemolytic anemia in adults

Agios Pharmaceuticals Inc. (NSDQ:AGIO) has announced that Pyrukynd (mitapivat) has won FDA approval to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

A first-in-class oral PK activator, mitapivat is the first disease-modifying therapy for hemolytic anemia to win FDA approval.

FDA based its decision on data from the Phase 3 ACTIVATE and ACTIVATE-T studies. Mitapivat met its primary endpoint in both studies.

Last year, Agios announced that 37% of patients who received mitapivat in the ACTIVATE trial achieved at least 33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks.

To maximize the availability of Pyrukynd, Agios is offering select patients $0 copays for the medication. The company also has a patient assistance program (PAP) that provides free prescriptions to certain uninsured and underinsured patients.

“For more than a decade, we have been pioneering th…

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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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