FDA Approves Agios Drug For Thalassemia-Associated Anemia

The U.S. Food and Drug Administration (FDA) on Tuesday approved Agios Pharmaceuticals, Inc.’s (NASDAQ:AGIO) Aqvesme (mitapivat), an oral pyruvate kinase (PK) activator, for anemia in adults with alpha- or beta-thalassemia, a rare type of blood disorder that impacts hemoglobin production.

With this approved indication, Aqvesme becomes the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

The FDA approval is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively.

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A total of 452 patients who are representative of the real-world thalassemia population were enrolled in the trials.

Efficacy Outcomes And Quality-Of-Life Benefits

The ENERGIZE and ENERGIZE-T Phase 3 trials met all primary and key secondary efficacy endpoints, demonstrating that Aqvesme improves hemolytic anemia and a key quality-of-life measure compared to placebo, including significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.

In the ENERGIZE and ENERGIZE-T Phase 3 trials, five patients receiving Aqvesme experienced adverse reactions suggestive of hepatocellular injury (HCI), with two of these patients requiring hospitalization.

These adverse reactions occurred within the first six months of exposure, and liver tests improved upon discontinuation of Aqvesme.

REMS Program And Labeling Strategy

To mitigate the risk of HCI, Aqvesme is available only through the Aqvesme REMS, a Risk Evaluation and Mitigation Strategy (REMS) program approved by the FDA.

The Aqvesme REMS program requires liver tests before the first AQVESME dose, every four weeks thereafter for 24 weeks, and then as clinically indicated.

Due to the Aqvesme REMS program, mitapivat will be marketed under the brand name Aqvesme in the U.S. for the thalassemia indication.

Mitapivat will continue to be marketed as Pyrukynd (mitapivat) in the U.S. for the PK deficiency indication, which does not require a REMS program.

Global Branding And Commercial Timeline

Outside the U.S., mitapivat will continue to be marketed as Pyrukynd for its PK deficiency and thalassemia indications in regions where it is approved, and will retain this name upon approval in regions currently under regulatory review.

Agios expects Aqvesme to be available in the U.S. in late January 2026, following implementation of the Aqvesme REMS program.

AGIO Price Action: Agios Pharmaceuticals shares were up 9.80% at $27.00 during premarket trading on Wednesday, according to Benzinga Pro data.

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