AGIO's Mitapivat Gets FDA Nod for Thalassemia Indication with REMS in Label

Core Insights - Agios Pharmaceuticals (AGIO) received FDA approval for Aqvesme (mitapivat) to treat adult patients with alpha- or beta-thalassemia, making it the only FDA-approved drug for both non-transfusion-dependent and transfusion-dependent forms of the disease [1][6] - The launch of Aqvesme is anticipated in late January 2026, following the implementation of the AQVESME REMS program [2][6] - The approval is based on phase III ENERGIZE and ENERGIZE-T studies, which showed significant improvements in hemoglobin levels, fatigue, and reduced blood transfusion needs compared to placebo [3][6] Regulatory and Market Context - The FDA's approval included a Risk Evaluation and Mitigation Strategy (REMS) due to reported cases of hepatocellular injury among treated patients, which may have negatively impacted investor sentiment, leading to a 1.5% decline in AGIO's stock [4][6] - AGIO's shares have decreased by 28.1% over the past year, while the industry has seen a rise of 16.8% [5] Future Developments - Agios has filed for regulatory approval of Aqvesme in the European Union, with a positive opinion from the EMA expected to lead to a final decision in early 2026 [9] - Outside the U.S., mitapivat will continue to be marketed as Pyrukynd for both PK deficiency and thalassemia indications, with ongoing studies for sickle cell disease [10]