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 deficiency can result in a range of symptoms, including chronic fatigue, hemolytic crisis, iron overload, gallstones, splenomegaly, ulcers, liver cirrhosis, pulmonary hypertension and osteoporosis
The Cambridge, Mass.–based company filed the NDA based on ACTIVATE and ACTIVATE-T pivotal studies.
Agios Pharmaceuticals also plans to submit a marketing authorization application for mitapivat in the European Union in mid-2021.
Filed Under: clinical trials, Drug Discovery
