Agios Pharmaceuticals Says It Will Pursue US Accelerated Approval For Mitapivat For Sickle Cell Disease

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) announced its intention to pursue U.S. accelerated approval for mitapivat, an oral pyruvate kinase activator for sickle cell disease, following a pre-sNDA meeting with the FDA. The FDA recommended that Agios submit a proposal for a confirmatory clinical trial to support this accelerated approval, which is designed to expedite access to treatments for serious conditions. Agios has already submitted this proposal, which includes a primary endpoint different from those in previous trials, and plans to submit the sNDA in the coming months while maintaining its operating expense guidance through 2025.