Credit: Shutterstock. Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes. The Food and Drug Administration (FDA) has accepted for Priority ...

Pyruvate Kinase Deficiency Impact Assessment (PKDIA), a weekly measure of disease impacts, measures health-related quality of life, (e.g., daily activities, concentration, physical activity and the ...

— RP-A501 for Danon Disease well-tolerated in two pediatric patients with no complement-mediated toxicities reported; efficacy update remains on track for Q3 — — Sustained genetic correction observed ...

PDUFA goal date extended by three months from September 7, 2025, to December 7, 2025CAMBRIDGE, Mass., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Agios ...

Results from a phase 3 clinical indicate that the oral drug mitapivat is safe and effective for treating adults with pyruvate kinase deficiency, a genetic condition that causes red blood cell ...

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) announced Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion on ...

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare ...

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, ...

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare ...

The FDA approved mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. Pyruvate kinase (PK) deficiency is a rare inherited disorder that causes anemia via the ...

Pyruvate kinase deficiency, the most common genetic lesion in the glycolytic pathway, leads to chronic hemolytic anemia. Mitapivat, an oral agent, can activate some mutant enzymes and restore red-cell ...