Agios submits NDA to the FDA for mitapivat for treatment of adults with pyruvate kinase deficiency.

“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 Chris Bowden, M.D., chief medical officer at Agios. “There are no approved therapies for PK deficiency, and the current management strategies of blood transfusions and splenectomy are associated with both short- and long-term risks, including iron overload, blood clots and increased risk for infections. PK deficiency can lead to chronic fatigue, hemolytic crisis, gallstones, splenomegaly, liver cirrhosis, pulmonary hypertension and osteoporosis, and the burden of disease can take a toll on patients’ ability to navigate work and other day-to-day activities, as well as on their mental health. We are looking forward to working with the FDA to provide a potential new treatment option for this community that may help address the significant unmet needs of PK deficiency patients.”

The NDA submission is based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. A full analysis of these data – including patient-reported outcomes (PRO) – was recently presented at the European Hematology Association (EHA) Virtual Congress. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat. The company remains on track to submit a marketing authorization application (MAA) in the EU in mid-2021 for mitapivat in adults with PK deficiency.