Agios Pharmaceuticals has secured approval for PYRUKYND (mitapivat) from the US Food and Drug Administration (FDA) for the treatment of hemolytic anemia in adult patients having pyruvate kinase (PK) deficiency.

PYRUKYND is an oral pyruvate kinase activator, which is now the first approved disease-modifying therapy for the rare, debilitating, lifelong hemolytic anemia.

The FDA approval for PYRUKYND was driven by the findings of the ACTIVATE and ACTIVATE-T clinical trials held in not regularly transfused and regularly transfused adults with deficiency of pyruvate kinase, respectively.

The ACTIVATE phase 3 trial of mitapivat met its primary endpoint with the drug candidate delivering a statistically significant increase in hemoglobin in patients having deficiency of pyruvate kinase and who are not transfused regularly.

The ACTIVATE-T phase 3 trial of mitapivat also met its primary endpoint with oral pyruvate kinase activator showing a statistically significant and clinically meaningful decrease in transfusion burden for patients with regular transfusion.

Jackie Fouse — CEO of Agios Pharmaceuticals said: “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.

“We remain committed to partnering with patients, caregivers, advocates and healthcare providers to ensure that the impact of PYRUKYND is maximized through robust support, education and access programs. These connections have fueled today’s tremendous milestone for the PK deficiency community.”

  Agios Pharmaceuticals, anemia, Blood disorder, hemolytic anemia, Jackie Fouse, mitapivat, PYRUKYND, pyruvate kinase, US FDA, US Food and Drug Administration