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…