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Business Insider4y

Agios Announces Updated Data from ACTIVATE and ACTIVATE-T Phase 3 Studies of Mitapivat in Pyruvate Kinase (PK) Deficiency at the European Hematology Association Virtual Congress

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 ...
Business Wire3y

Rocket Pharmaceuticals Presents Positive Clinical Data from Danon Disease, Fanconi Anemia and Pyruvate Kinase Deficiency Programs at the 25th Annual Meeting of the American ...

— 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 ...
Business Insider4y

Agios to Present Updated Data from Clinical Trials of Mitapivat in Pyruvate Kinase Deficiency and Thalassemia in Oral Presentations at the European Hematology Association ...

– Agios to Host Investor Webcast on June 11 at 7:30 a.m. ET – CAMBRIDGE, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism ...
Science Daily3y

Phase 3 clinical trial results lead to approval of oral drug for red blood cell disorder

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 ...
Monthly Prescribing Reference3y

Pyrukynd Approved for Hemolytic Anemia Due to Pyruvate Kinase Deficiency

The FDA has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. The Food and Drug Administration (FDA) has approved Pyrukynd ® (mitapivat) ...
Nasdaq2y

Agios Says CHMP Adopts Positive Opinion For PYRUKYND To Treat Pyruvate Kinase Deficiency In Adults

(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 ...
FOX8 Cleveland3y

Agios Announces Publication of Phase 3 ACTIVATE-T Data in The Lancet Haematology Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase 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, ...
Business Wire2y

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

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 ...
TMCnet9d

Agios Provides Update on U.S. PDUFA Goal Date for PYRUKYND® (mitapivat) in Thalassemia

The PYRUKYND sNDA is supported by results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with NTD and TD alpha- or beta-thalassemia, ...
Healio3y

FDA approves Pyrukynd for adults with pyruvate kinase deficiency-related hemolytic anemia

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 ...
The New England Journal of Medicine3y

Mitapivat vs. Placebo for Pyruvate Kinase Deficiency

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 ...