RegulatoryMay 22, 2026, 09:17 AM
Agios PYRUKYND Approved in EU for Thalassemia
AI Summary
Agios Pharmaceuticals announced that the European Commission (EC) has granted marketing authorization for PYRUKYND (mitapivat) for adults with alpha- or beta-thalassemia. This approval makes PYRUKYND the only medicine approved in all EU member states for this broad patient population, addressing a significant unmet need. The decision is based on positive results from the global Phase 3 ENERGIZE and ENERGIZE-T trials. Mitapivat is now approved for thalassemia in the U.S., Saudi Arabia, United Arab Emirates, and the EU, with Avanzanite Bioscience B.V. handling European commercialization.
Key Highlights
- The European Commission granted marketing authorization for PYRUKYND (mitapivat) for adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.
- PYRUKYND is now the only medicine approved in all European Union member states for this broad patient population.
- Mitapivat is approved for thalassemia in the U.S., Saudi Arabia, United Arab Emirates, and the EU.
- Avanzanite Bioscience B.V. will continue to distribute and commercialize PYRUKYND across the European Economic Area, UK, and Switzerland.
- The EC's decision is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials.
- An estimated 18,000 to 23,000 children and adults are living with thalassemia in the U.S. and five largest European countries.
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