Monthly Prescribing Reference

Mitapivat Granted Priority Review for Pyruvate Kinase Deficiency

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 ...
Monthly Prescribing Reference

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) ...
Nasdaq

Agios Announces Publication of Phase 3 ACTIVATE Study in New England Journal of Medicine Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase Deficiency

– In Adults with Pyruvate Kinase (PK) Deficiency Who Are Not Regularly Transfused, PYRUKYND® Significantly Increased Hemoglobin Level, Decreased Hemolysis and Improved Patient-Reported Outcomes – – ...
Business Wire

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

Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral-Mediated Gene Therapy: Interim Results From an Ongoing Global Phase 1 Study The poster presentation includes data ...
Nasdaq

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 ...
Business Insider

Agios Establishes Proof-of-Concept for Mitapivat in Non-transfusion-dependent Thalassemia Based on Preliminary Phase 2 Results

– Treatment with Mitapivat Induced Hemoglobin Increase of ≥1.0 g/dL in 7 of 8 Evaluable Patients – – Safety Profile Consistent with Previously Published Phase 2 Data for Mitapivat in Patients with ...
Healio

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 ...
WHNT

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, ...
Science Daily

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 ...
Business Wire

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 ...
FOX31 Denver

PYRUKYND® (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients

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 ...
The Boston Globe

Agios wins FDA approval for pill to treat rare blood disease

The Cambridge company’s CEO says the medicine for people with pyruvate kinase deficiency has the potential to become a blockbuster drug. Federal regulators have approved the first medicine for people ...