India, May 12 -- Rare disease drugmaker Agios Pharmaceuticals Inc. (AGIO) on Tuesday announced the submission of a supplemental New Drug Application, or sNDA, to the U.S. Food and Drug Administration seeking accelerated approval of mitapivat for the treatment of sickle cell disease.
The filing follows agreement with the FDA on a confirmatory clinical trial required under the accelerated approval pathway.
The confirmatory trial will evaluate mitapivat's ability to reduce transfusion burden in patients with sickle cell disease and is expected to enroll about 159 patients aged 12 years and older.
Agios said the sNDA is supported by data from the Phase 2 and Phase 3 RISE UP clinical program, which demonstrated statistically significant impro...