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# Agios Filing NDA on Thalassemia Drug
- URL: https://www.fdaweb.com/agios-filing-nda-on-thalassemia-drug/
- Published: 2024-06-03T12:00:00.000Z
- Updated: 2026-09-14T14:32:20.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157100

Agios Pharmaceuticals plans to submit by the end of the year an NDA for mitapivat and its use in adults with transfusion-dependent alpha- or beta-thalassemia. The move is based on just-announced Phase 3 (ENERGIZE-T) study data that met the primary endpoint of transfusion reduction response. “Statistical significance was also achieved for all key secondary endpoints evaluating additional measures of reduction of transfusion burden compared to placebo,” the company says.

“Treatment options for patients with transfusion-dependent thalassemia are limited, and transfusions carry significant risks for patients, such as iron overload and immune reactions,” a company release says. “Based on these data demonstrating that treatment with mitapivat significantly reduces transfusion burden across alpha- and beta-thalassemia patients, along with its convenient oral formulation, mitapivat has the potential to become a novel advancement in care for thalassemia patients.”

Agios says topline results from the ENERGIZE-T showed:

- The study met its primary endpoint of a 50% reduction or more in transfused red blood cell units in any consecutive 12-week period).
- Treatment with mitapivat demonstrated a statistically significant reduction in transfusion burden compared to placebo, as measured by the transfusion reduction response (TRR) endpoint. In the mitapivat arm, 30.4% of patients achieved a TRR, compared to 12.6% of patients in the placebo arm (2-sided p=0.0003).
- Overall, during the 48-week double-blind period, incidence of adverse events (AEs) was similar across mitapivat and placebo arms. In the mitapivat arm, 5.8% of the patients experienced an AE leading to discontinuation, compared to 1.2% of patients in the placebo arm.