FDA Approves Mitapivat for Thalassemia Anemia; Vadadustat Dosing Trial Meets Safety Goals
The FDA has approved Agios Pharmaceuticals' mitapivat (AQVESME) as the first oral treatment for anemia in thalassemia, reducing transfusion needs by 40-50%. Meanwhile, a clinical trial found a three-times-weekly dosing of vadadustat (Vafseo) is safe for kidney dialysis patients, potentially improving convenience.
The U.S. Food and Drug Administration has approved the first-ever oral pill to treat anemia in adults with thalassemia, while a separate clinical trial has demonstrated that a less frequent dosing schedule of another oral anemia drug is safe for patients on kidney dialysis.
Developed by Agios Pharmaceuticals, AQVESME (mitapivat) is a first-in-class oral pyruvate kinase (PK) activator designed to improve red blood cell energy balance, reduce hemolysis, and decrease transfusion dependency. The approval was granted based on data from the global Phase 3 Energize and Energize-T trials, which evaluated its efficacy in both non-transfusion-dependent and transfusion-dependent alpha and beta thalassemia. In clinical trials, mitapivat demonstrated a 40-50% rate in reducing transfusion requirements among adults with thalassemia. Unlike conventional therapies that primarily address symptoms, mitapivat targets the underlying metabolic dysfunction within red blood cells by activating the pyruvate kinase enzyme to stabilize red blood cells and reduce premature destruction. Experts note that while the drug has not yet been approved by the Drugs Controller General of India, its availability, combined with existing therapies like thalidomide and luspatercept, could significantly ease the treatment burden for thousands of thalassemia patients in India.
In a separate development, a clinical trial led by UC Davis Health faculty found that a three-times-weekly dosing schedule of vadadustat (Vafseo) appears safe for patients undergoing dialysis. Vadadustat, an oral medication, was approved by the FDA in 2024 for once-daily use to treat anemia caused by chronic kidney disease in adults. The new trial enrolled over 2,000 patients at more than 160 dialysis facilities nationwide and compared the three-times-weekly oral treatment with standard injectable therapy. The study met its primary goal of demonstrating that the oral treatment was at least as safe as standard therapy, and an independent monitoring committee recommended ending the study early due to the favorable safety profile. A UC Davis Health kidney specialist noted that aligning treatment with regular dialysis sessions could improve adherence and reduce the burden of managing an additional at-home medication.