AMSTERDAM--(BUSINESS WIRE)-- #Avanzanite--Avanzanite Bioscience B.V., a rapidly growing commercial-stage European specialty pharmaceutical company focused on rare diseases, today reported that its partner, Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts focused on delivering innovative medicines for patients with rare diseases, announced that the European Commission has granted marketing authorisation for PYRUKYND® (mitapivat).

PYRUKYND is the only medicine approved in the EU for adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia Mitapivat is now approved for thalassemia in the U.S., Saudi Arabia, United Arab Emirates, and EU Avanzanite Bioscience B.V., a specialty pharmaceutical company, will continue to distribute and commercialize PYRUKYND in Europe CAMBRIDGE, Mass.

Agios Pharmaceuticals NASDAQ: AGIO executives said the company is focused on advancing mitapivat across rare hematologic diseases, highlighting early launch traction in thalassemia and a recently submitted supplemental new drug application for sickle cell disease during a presentation at the 2026 RBC Capital Markets Global Healthcare Conference.

AGIO seeks FDA accelerated approval for mitapivat in sickle cell disease after mixed phase III data and a planned confirmatory study.

sNDA submission follows agreement with FDA on confirmatory trial, a requirement of the accelerated approval pathway Confirmatory trial designed to demonstrate clinical benefit of mitapivat on reducing transfusion burden in sickle cell disease CAMBRIDGE, Mass. , May 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.

sNDA submission follows agreement with FDA on confirmatory trial, a requirement of the accelerated approval pathway Confirmatory trial designed to demonstrate clinical benefit of mitapivat on reducing transfusion burden in sickle cell disease CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the U.S. accelerated approval of mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease.

Detailed results from RISE UP Phase 3 trial of mitapivat in sickle cell disease selected for distinguished Plenary Abstracts Session Company to host investor conference call and webcast during EHA 2026 on Saturday, June 13, at 9:00 a.m. ET (3:00 p.m.

The company faces competitive pressure from Novo Nordisk, but it's growing sales from its existing approved drugs and has an exciting drug in its pipeline.

Agios shares jump 13% as Q1 revenues surge 138% on Aqvesme launch and Pyrukynd growth, while pipeline advances and SCD filing plans take shape.

There's power in the company's No. 1 molecule.