Agios Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for PYRUKYND® (mitapivat) for the treatment of pyruvate kinase (PK) deficiency in adult patients.
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy for European patients with PK deficiency.
“The positive CHMP opinion for PYRUKYND® is a landmark event for European patients with PK deficiency, who currently have no disease-modifying treatment options,” said Eduard J. van Beers, M.D., Ph.D., Haematologist and Associate Professor at University Medical Center Utrecht. “PK deficiency is characterized by severe symptoms and complications, regardless of transfusion status, and has a significant impact on patients’ quality of life. If approved, I look forward to having an oral medication available that has demonstrated clinically meaningful impact for patients with a range of PK deficiency symptoms and presentations.”
“Today’s positive CHMP opinion is an important step toward our goal of expanding PYRUKYND® access for adults with PK deficiency around the world,” said Sarah Gheuens, M.D., Ph.D., head of R&D and chief medical officer at Agios Pharmaceuticals. “In addition to this milestone, we remain focused on our global clinical development programmes for PYRUKYND® in thalassemia, sickle cell disease and pediatric PK deficiency and look forward to expanding the impact of this medication to even more patients.”
PYRUKYND® was previously granted orphan drug designation by the EMA.