Agios Pharmaceuticals Inc. (NASDAQ: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 the science of PK activation in order to bring Pyrukynd to people with PK deficiency and provide them with the first medication approved specifically to address this rare, debilitating blood disorder,” Agios CEO Jackie Fouse said in a statement.
AGIO shares fell 5.18% to $29.46.
Filed Under: clinical trials, Drug Discovery, Hematology