Agios’ PYRUKYND® (mitapivat) approved by UAE’s EDE as first therapy for adult alpha/beta-thalassemia (transfusion-dependent & non-dependent). Phase 3 ENERGIZE/ENERGIZE-T data support hemoglobin response & transfusion reduction in GCC region.
Written By: Sana Khan BPharm
Reviewed By: Pharmacally Editorial Team
Agios Pharmaceuticals, Inc. announced a major breakthrough for thalassemia treatment in the United Arab Emirates (UAE). The Emirates Drug Establishment (EDE) has approved PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, for adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. This makes PYRUKYND the only approved medicine in the UAE for this patient population, addressing a long-standing gap in care.
Thalassemia, a rare inherited blood disorder, impairs hemoglobin production, leading to anemia, fatigue, and life-threatening complications. In the Gulf Cooperation Council (GCC) region including Saudi Arabia, UAE, Kuwait, Qatar, Oman, and Bahrain around 70,000 people live with the disease.
“For too long, thalassemia patients have endured debilitating symptoms without significant innovation,” said Khaled Musallam, M.D., Ph.D., from Burjeel Medical City in Abu Dhabi and an investigator in the Phase 3 program. He emphasized PYRUKYND’s potential to tackle these challenges, especially given the high regional prevalence.
The approval draws from robust data in the global Phase 3 ENERGIZE (NCT04770753) and ENERGIZE-T (NCT04770779) trials. ENERGIZE enrolled 194 non-transfusion-dependent adults, meeting its primary endpoint of hemoglobin response (≥1.0 g/dL increase from baseline over Weeks 12-24).
ENERGIZE-T involved 258 transfusion-dependent adults, achieving its primary endpoint of transfusion reduction response (≥50% reduction in RBC units over 12-week periods through Week 48).
Both trials confirmed safety and tolerability, with open-label extensions available for participants.
“This milestone underscores our commitment to rare blood disorders,” said Brian Goff, CEO of Agios. The company partnered with NewBridge Pharmaceuticals in 2024 to drive regulatory and commercial efforts across the GCC. PYRUKYND is already launching in Saudi Arabia following SFDA approval in August 2025, building regional momentum. Agios is also advancing mitapivat in the Phase 3 RISE UP trial for sickle cell disease, further expanding its potential in rare blood disorders.
Experts hailed the news. Androulla Eleftheriou, Ph.D., Executive Director of Thalassaemia International Federation, called it a “pivotal moment” that expands options for patients with limited prior treatments. PYRUKYND is approved for PK deficiency in the US and Europe, with a thalassemia application under review by the European Commission. In the US, mitapivat is also approved for thalassemia as AQVESME™.
For full prescribing information on PYRUKYND, including indications, safety, and usage details, visit PYRUKYND Prescribing Information. For AQVESME, see AQVESME Prescribing Information.
References
Agios’ PYRUKYND® (mitapivat) Approved for Adults with Thalassemia in the United Arab Emirates, Mar 02, 2026. https://investor.agios.com/news-releases/news-release-details/agios-pyrukyndr-mitapivat-approved-adults-thalassemia-united
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT), ClinicalTrials.gov ID NCT04770753. https://clinicaltrials.gov/study/NCT04770753
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T), ClinicalTrials.gov ID NCT04770779. https://clinicaltrials.gov/study/NCT04770779
About Writer
Sana Jamil Khan, B.Pharm
She is a pharmacy graduate with a keen interest in clinical research, pharmacovigilance, and medical writing, with a growing focus on publication and scientific content development. In her words, she is passionate about translating complex medical data into clear, evidence-based communication.