Agios submits sNDA to FDA for accelerated approval of mitapivat in sickle cell disease.

Agios Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to the FDA seeking accelerated approval for mitapivat, an oral treatment for sickle cell disease. This submission follows an agreement with the FDA on a confirmatory trial designed to show mitapivat's benefit in reducing transfusion needs. The confirmatory trial will enroll about 159 patients aged 12 and older and aims to demonstrate clinical benefits over 52 weeks. Agios expects FDA review acceptance by Q3 2026, potentially making mitapivat the first pyruvate kinase activator approved in the U.S. for sickle cell disease, addressing a significant unmet medical need.