newsfilter
← Latest News
GlobeNewswire·

Agios' PYRUKYND® (mitapivat) Approved in the European Union for Adults with Thalassemia

AGIO
  • The European Commission granted marketing authorization for PYRUKYND (mitapivat) to treat anemia in adults with transfusion-dependent & non-transfusion-dependent alpha- or beta-thalassemia.
  • PYRUKYND is the only medicine approved in all EU member states for this specific patient population.
  • Mitapivat is now approved for thalassemia in the U.S., Saudi Arabia, United Arab Emirates, & the EU.
  • Agios Pharmaceuticals announced that Avanzanite Bioscience B.V. will continue to distribute & commercialize PYRUKYND in Europe under a 2025 exclusive agreement.
  • The EC approval was based on positive results from the global, randomized, double-blind, placebo-controlled ENERGIZE & ENERGIZE-T Phase 3 clinical trials.
  • The ENERGIZE trial focused on non-transfusion-dependent patients, while the ENERGIZE-T trial focused on transfusion-dependent patients.
  • In the U.S., the medicine is marketed as AQVESME™ for thalassemia & PYRUKYND for PK deficiency.
  • AQVESME carries a Boxed Warning for hepatocellular injury & is available only through a restricted REMS program.
  • PYRUKYND carries safety warnings regarding acute hemolysis upon abrupt discontinuation & potential liver injury in patients with other conditions treated at higher doses.
  • Approximately 18,000 to 23,000 children & adults live with thalassemia in the U.S. & the five largest European countries.