Mitapivat

Unassigned

New Medicines

Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults

Information

New molecular entity
Agios Pharmaceuticals
Agios Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

First-in-class, small molecule stimulator of pyruvate kinase
Pyruvate kinase deficiency (PKD) occurs worldwide but most cases have been reported in northern Europe, Japan and the USA. Many cases are found in the Amish population of Pennsylvania. The prevalence is estimated at 51 cases per million by gene frequency studies but the observed prevalence in one northern England region was found to be 3.3 cases per million [1].
Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults
Oral