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Agios’ PYRUKYND® (mitapivat) Approved for Adults with Thalassemia in the United Arab Emirates
PYRUKYND is the only medicine approved in the UAE for adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia NewBridge
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"PYRUKYND is the only medicine approved in the UAE for adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia NewBridge Pharmaceuticals, a regional specialty company, will continue to manage commercialization of PYRUKYND in the Gulf region CAMBRIDGE, Mass., March 02, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the Emirates Drug Establishment (EDE) of the United Arab Emirates (UAE) has approved PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. With this approval, PYRUKYND becomes the only medicine approved in the UAE for this patient population. “For too long, thalassemia patients have endured debilitating and often life-threatening symptoms in the absence of significant therapeutic innovation,” said Khaled Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, UAE, and an investigator in the PYRUKYND thalassemia Phase 3 clinical program. “The robust Phase 3 results demonstrate that PYRUKYND can meet this urgent need by addressing the complex challenges of thalassemia. Given the high prevalence of the disease in the Gulf region, today’s approval marks a critical milestone, offering thalassemia patients in the UAE – regardless of genotype or transfusion burden – a new treatment option to help manage their disease.” The EDE approval of PYRUKYND in thalassemia is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. “Agios is steadfast in our dedication to advancing innovative medicines for patients with rare blood disorders,” said Brian Goff, Chief Executive Officer, Agios. “Today’s milestone underscores this commitment while highlighting the transformative potential of PYRUKYND as a disease-modifying oral therapy for thalassemia. Together with our partner NewBridge Pharmaceuticals, we look forward to bringing this novel treatment option to patients in the UAE.”In 2024, Agios entered into a distribution agreement with NewBridge Pha...