Agios Pharmaceuticals said Monday that its pill Pyrukynd increased levels of hemoglobin in more sickle cell disease patients than placebo, and it plans to start a Phase III trial later this year.
New drug approvals were fewer in number in 2022. Is this a trend that will continue into 2023 or is the drop off just a cyclical anomaly?
Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH Annual Meeting and Exposition
CAMBRIDGE, Mass., Dec. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported new data from the ongoing long-term extension period of the Phase 2 open-label study of PYRUKYND® (mitapivat), a first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with non-transfusion dependent ?- or ?-thalassemia. Data from the study were featured in a poster presentation (abstract #1030) at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, hosted Dec. 10-13, 2022, in New Orleans.
CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, today announced that the European Commission (EC) has granted marketing authorization for PYRUKYND® for the treatment of PK deficiency in adult patients. PYRUKYND® is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the EU with this rare, debilitating, lifelong hemolytic anemia.
CAMBRIDGE, Mass., Sept. 19, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, today announced the appointment of Cecilia Jones to the role of chief financial officer, effective Sept. 26, 2022. Ms. Jones will replace Jonathan Biller, who served as the company’s previous chief financial officer and head of corporate affairs and who resigned effective Sept. 16, 2022 in order to become chief legal officer of Vertex Pharmaceuticals.
CAMBRIDGE, Mass., Sept. 16, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion on September 15, 2022, recommending the granting of a marketing authorization for PYRUKYND® (mitapivat) for the treatment of pyruvate kinase (PK) deficiency in adult patients. PK deficiency is a rare, debilitating, lifelong hemolytic anemia. PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy for European patients with PK deficiency.
CAMBRIDGE, Mass., Aug. 18, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced that data from the core period of the pivotal Phase 3 ACTIVATE-T study of PYRUKYND® (mitapivat) in adults with pyruvate kinase (PK) deficiency who receive regular transfusions were published on August 18, 2022, in The Lancet Haematology. Data from this study were previously presented at the 2021 European Hematology Association (EHA) Annual Congress. PYRUKYND® is a first-in-class, oral PK activator and the first and only approved disease-modifying treatment for this rare, debilitating, lifelong hemolytic anemia.
Agios Announces Publication of Ph2 Data in The Lancet Safety of PYRUKYND®