Agios Pharmaceuticals (NASDAQ:AGIO) shares climbed about 12% on Tuesday after the company reported progress toward a potential accelerated U.S. approval for its drug candidate mitapivat in treating sickle cell disease following a pre-supplemental New Drug Application meeting with the U.S. Food and Drug Administration.
During the discussions, the FDA advised Agios to submit a proposal outlining a confirmatory clinical trial that could support an accelerated approval pathway for mitapivat, an oral pyruvate kinase activator. Accelerated approval allows treatments addressing serious conditions with unmet medical needs to reach patients sooner, provided that additional studies later confirm the drug’s clinical benefit.
Agios said it has already submitted its proposal for the required confirmatory trial to the FDA for review. The design includes a primary endpoint different from those used in the RISE UP clinical program, drawing on data analyses from the program as well as regulatory discussions.
The company plans to file a supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease in the coming months. It is also continuing discussions with the FDA to align on the details of the confirmatory trial needed to support the submission.
Based on its current plans, Agios said the proposed study is not expected to alter its previously issued operating expense outlook, which remains projected to be roughly flat compared with 2025.
The recent regulatory meeting focused on data from the RISE UP clinical program, which includes both Phase 2 and Phase 3 trials evaluating mitapivat in patients with sickle cell disease.