Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases,  announced that the European Commission has adopted a positive decision for the designation of mitapivat, an oral, small molecule PK activator, as an orphan medicinal product (OMP) for the treatment of sickle cell disease. Earlier, in November 2020, the U.S. Food and Drug Administration (FDA) also granted orphan drug designation to mitapivat for sickle cell disease.

“Alongside the FDA’s orphan drug designation in the U.S., the European Commission’s orphan medicinal product designation for mitapivat underscores the urgent need for novel therapies for sickle cell disease and highlights its potential to provide clinically meaningful benefits to patients navigating this debilitating condition,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “With the trial fully enrolled, we look forward to sharing the results of our Phase 3 RISE UP study evaluating the efficacy and safety of mitapivat in sickle cell disease with the community in late 2025.”

The European Commission offers OMP designation to innovative therapies that address life-threatening or chronically debilitating conditions affecting fewer than five in 10,000 individuals in the European Union, and that have the potential to provide a significant benefit over existing treatments. This designation provides extensive benefits to encourage the development of these medicines, including reduced fees and a 10-year period of market exclusivity.