(RTTNews) - 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 improvement in hemoglobin response compared with placebo.
The company said patients receiving mitapivat who achieved hemoglobin response also experienced clinically meaningful reductions in pain crises, related hospitalizations, and fatigue.
However, the Phase 3 study's co-primary endpoint evaluating annualized sickle cell pain crisis rates did not achieve statistical significance.
The company expects to receive notice of filing acceptance and a review timeline from the FDA during the third quarter of 2026, following the agency's 60-day filing review process.
Mitapivat is an oral pyruvate kinase activator designed to improve red blood cell health by increasing ATP production and lowering levels of 2,3-DPG, a molecule associated with sickling of red blood cells.
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