NEW APPROVAL | PYRUKYND® Gets EU Green Light For Pyruvate Kinase (PK) Deficiency 

Pyrukynd (mitapivat) becomes the first approved disease-modifying therapy for patients in the EU with the rare, debilitating, lifelong hemolytic anemia, pyruvate kinase (PK) deficiency


The European Commission has granted, on November 10, 2022, marketing authorization to Agios Pharmaceuticals for PYRUKYND® intended for the treatment of PK deficiency in adult patients. People with PK deficiency suffer from a lifetime of chronic anemia, associated complications and symptoms that can be detrimental to their work, family and social lives.

“With today’s EU approval, we are proud to expand the positive impact of PYRUKYND® for more patients with PK deficiency around the globe,” said Brian Goff, Chief Executive Officer at Agios. “We are dedicated to continued innovation on behalf of people with rare and genetically defined diseases, and are working to further expand the impact of PYRUKYND® through our ongoing investigational pivotal programs in pediatric PK deficiency, thalassaemia and sickle cell disease.”

PYRUKYND® has already been approved by the U.S. Food and Drug Administration (FDA) in February 2022 for the treatment of hemolytic anemia in adults with PK deficiency. Agios has also applied for a marketing authorization for PYRUKYND® as a treatment for PK deficiency in adult patients in Great Britain, under the European Commission Decision Reliance Procedure (ECDRP) with the Medicines and Healthcare Products Regulatory Agency (MHRA).

Agios is providing access to PYRUKYND® for the treatment of PK deficiency in adults receiving care in the EU through a global managed access program. More details about this program can be found on

The company also continues to advance its Phase 3 ENERGIZE and ENERGIZE-T studies in non-transfusion-dependent and transfusion-dependent adults with thalassemia, respectively. Learn more about these clinical trials here.

Read the Full Press Release on the EU approval of Mitapivat HERE

Back to top button