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Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat in Pyruvate Kinase Deficiency
CAMBRIDGE, Mass., March 30, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., March 30, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the company’s application for orphan drug designation for its investigational medicine mitapivat as a potential treatment for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia. Mitapivat was previously granted orphan drug designation by the United States Food and Drug Administration. Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes that directly targets the underlying metabolic defect in PK deficiency.\n “We are pleased to receive a positive opinion from EMA for orphan drug designation for mitapivat as a recognition of the need for innovative treatments for patients with PK deficiency, a serious lifelong disease characterized by hemolytic anemia that can lead to severe symptoms including iron overload, splenomegaly and heart failure,” said Chris Bowden, M.D., chief medical officer at Agios. “Both of our pivotal trials are fully enrolled, and we look forward to the completion of these trials intended to support global regulatory filings. Mitapivat is the first and only PKR activator to receive this important designation in Europe and the first potential disease-modifying treatment for PK deficiency patients.” Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member state...