Agios Secures UAE Approval for Mitapivat in Transfusion and Non-Transfusion Thalassemia
The Emirates Drug Establishment approved mitapivat for adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in the UAE, making it the only licensed therapy for this population. NewBridge Pharmaceuticals will manage Gulf commercialization under a 2024 distribution agreement spanning six GCC countries.
1. UAE Approval Details
The Emirates Drug Establishment granted marketing authorization for mitapivat to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. This designation makes mitapivat the sole approved therapy for these thalassemia subtypes in the UAE, addressing both genotypes and transfusion burdens.
2. Clinical Trial Evidence
Approval was based on Phase 3 ENERGIZE and ENERGIZE-T trials, which randomized 194 non-transfusion-dependent and 258 transfusion-dependent patients to mitapivat 100 mg twice daily or placebo. Primary endpoints included ≥1 g/dL hemoglobin increases and ≥50% reduction in red blood cell transfusion units, both of which were met with statistical significance.
3. Gulf Commercialization Strategy
Under a 2024 agreement, NewBridge Pharmaceuticals will oversee launch and distribution across the six-member Gulf Cooperation Council, including Saudi Arabia, UAE, Kuwait, Qatar, Oman and Bahrain. The company is already preparing commercial operations in Saudi Arabia following the August 2025 approval.
4. Broader Regulatory Outlook
Mitapivat is also approved for PK deficiency in the U.S. and EU, with a marketing application for thalassemia under review by European regulators. Agios aims to leverage this Middle East milestone as a template for further label expansions and regional partnerships.