AMSTERDAM — Avanzanite Bioscience said partner Agios Pharmaceuticals has received European Commission approval for PYRUKYND, an oral pyruvate kinase activator, to treat anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia in adults.
The approval gives PYRUKYND orphan medicinal product designation for the thalassaemia indication and marks the drug’s second approved use in the European Union. It was previously approved in 2022 for adults with pyruvate kinase deficiency.
Avanzanite, a commercial-stage European specialty pharmaceutical company focused on rare diseases, will commercialize and distribute PYRUKYND in Europe under an exclusive agreement with Cambridge, Massachusetts-based Agios.
“Thalassaemia is a complex, chronic and multisystem disease characterised by anaemia, ineffective erythropoiesis and haemolysis, which together place a significant burden on patients, including a substantial impact on quality of life and persistent fatigue,” said Raffaella Origa M.D., PhD., Professor of Paediatrics at University of Cagliari, Italy and President of the Italian Society of Thalassaemia and Haemoglobinopathies (SITE). “The approval of PYRUKYND in the EU represents an important step forward, introducing a new oral treatment option regardless of genotype or transfusion burden, with the potential to address key aspects of the disease, including reducing transfusion burden and improving patient outcomes.”
The European Commission’s decision followed a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use. The approval was based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials.
“The treatment of thalassaemia continues to place a profound burden on patients and healthcare systems,” said Antonis Kattamis M.D., Professor at the National & Kapodistrian University of Athens, Greece, and an investigator in the PYRUKYND thalassaemia Phase 3 clinical program. “An oral therapy such as PYRUKYND has the potential to transform the care of both transfusion-dependent and non-transfusion-dependent patients, and we welcome being able to provide this option in our clinical practice.”
Avanzanite entered into its exclusive agreement with Agios in June 2025 to commercialize and distribute PYRUKYND across the European Economic Area, the United Kingdom and Switzerland. The company said it will work with Agios, local health authorities and patient communities to support access across the EU.
“Today’s approval of Agios’ first-in-class PK activator in the EU is great news for adults living with thalassaemia and we are proud and privileged to partner with Agios to distribute and commercialise this medicine in the region,” said Adam Plich, CEO and Co-Founder of Avanzanite Bioscience. “Our role now is to collaborate with local authorities, drive a successful launch, and enable broad access to PYRUKYND in this indication, helping to ensure no thalassaemia patient is left behind, across the EU.”
Avanzanite said the approval represents the fourth rare disease launch it has led. The company said its commercial platform includes more than 100 rare disease professionals operating across 32 European countries.
