Agios’ Mitapivat Granted Orphan Status By European Union For Sickle Cell Disease

Agios Pharmaceuticals, Inc. is a pioneer in cellular metabolism and pyruvate kinase (PK) activation therapies for rare diseases. The company has announced that the European Commission has granted the designation of orphan medicinal product (OMP) to mitapivat. The drug is an oral PK activator for the treatment of sickle cell disease. This follows a similar orphan drug designation from the U.S. FDA in November 2020. 

Sarah Gheuens, M.D., PhD, chief medical officer and head of R&D at Agios, said in a statement, “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. 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 awards OMP designation to innovative treatments targeting severe or chronically debilitating conditions that impact fewer than 5 in 10,000 people in the EU and show promise for significant improvement over existing therapies. This designation provides key incentives, including reduced regulatory fees and 10 years of market exclusivity, to support the development of such medicines.