Wedoany.com Report-Dec.25, Agios Pharmaceuticals announced late on Tuesday that the U.S. Food and Drug Administration approved its drug mitapivat, branded as Aqvesme, for the treatment of anemia in adults with alpha- or beta-thalassemia. This includes both non-transfusion-dependent and transfusion-dependent forms of the condition.

The approval makes Aqvesme the only FDA-approved oral medicine for anemia in these patient groups. Thalassemia is an inherited blood disorder that impairs the body's ability to produce sufficient hemoglobin and healthy red blood cells.

Shares of Agios Pharmaceuticals rose significantly on Wednesday following the announcement, with gains reported in the range of 12% to 18% in premarket and early trading.

The company plans to make Aqvesme available in late January 2026, after implementing a required Risk Evaluation and Mitigation Strategy program. The label includes a boxed warning for potential hepatocellular injury and mandates liver function tests every four weeks for the first 24 weeks of treatment. It also advises against use in patients with cirrhosis. Analysts have noted that these safety measures align with expectations.

Chief Financial Officer Cecilia Jones stated during an analyst call on Wednesday that the annual wholesale acquisition cost for the drug is approximately $425,000 per patient.

Cantor analyst Eric Schmidt commented: "This is a modest premium to Pyrukynd's wholesale acquisition cost price of $335,000. We had expected parity pricing."

Mitapivat, marketed as Pyrukynd, received FDA approval in 2022 for treating hemolytic anemia in adults with pyruvate kinase deficiency.

Truist analyst Gregory Renza said: "The approval unlocks an additional $320 million in peak revenue opportunity layered atop the existing mitapivat franchise."

Agios targets an addressable population of about 4,000 adult thalassemia patients in the U.S. at launch, out of an estimated total of around 6,000 adults affected by the disorder.

The approval is supported by data from the global Phase 3 ENERGIZE and ENERGIZE-T trials, which enrolled 452 adult patients. These randomized, double-blind, placebo-controlled studies met all primary and key secondary endpoints, demonstrating statistically significant improvements in hemoglobin levels, reductions in transfusion requirements for dependent patients, and enhancements in fatigue and quality-of-life measures compared to placebo.

This development expands treatment options for individuals living with thalassemia, addressing key challenges such as anemia and related complications through an oral, disease-modifying therapy. Agios continues to focus on rare blood disorders, building on the established use of mitapivat in other indications.