Abu Dhabi, December 25, 2025: Research teams at Burjeel Holdings have played a significant role in advancing a major global milestone in thalassemia care, following the US Food and Drug Administration's (USFDA) approval of AQVESME™ (mitapivat).

The approval is supported by robust data from the global Phase 3 ENERGIZE and ENERGIZE-T clinical trials, conducted at Burjeel Cancer Institute’s Center for Research on Rare Blood Disorders and the Thalassemia & Sickle Cell Center. AQVESME™ is the first oral therapy indicated for anemia in adults with both non-transfusion-dependent and transfusion-dependent alpha- and beta-thalassemia.

The trials demonstrated clinically meaningful improvements in hemoglobin levels, reductions in transfusion burden, and better quality of life outcomes for patients living with these rare and lifelong blood disorders.

Commenting on the achievement, Prof. Khaled Musallam, Chief Research Officer at Burjeel Holdings and Principal Investigator of the trial at Burjeel, said, “This approval reflects years of disciplined global research and collaboration. Most importantly, it translates scientific progress into meaningful improvements in the lives of patients living with thalassemia.”

This milestone underscores Burjeel Holdings’ growing role in global clinical research and its commitment to translating scientific excellence into real-world patient impact.

🔗 For more details on the FDA approval: https://investor.agios.com/news-releases/news-release-details/us-fda-approves-agios-aqvesmetm-mitapivat-treatment-anemia

Burjeel Investigators Contribute to USFDA Approval of First Oral Therapy for Anemia in Alpha- and Beta-Thalassemia