> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Agios Seeks Expanded Use for Pyrukynd
- URL: https://www.fdaweb.com/agios-seeks-expanded-use-for-pyrukynd/
- Published: 2024-12-09T12:00:00.000Z
- Updated: 2026-09-14T14:48:53.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158327

Agios says it has filed a supplemental NDA for an expanded indication for its Pyrukynd (mitapivat) for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The pyruvate kinase activator is currently approved for treating hemolytic anemia in adults with pyruvate kinase deficiency.

The submission is based on recently reported data from the Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. The company says the data met its primary endpoint and demonstrated a statistically significant reduction in transfusion burden compared to placebo in patients with transfusion-dependent alpha- or beta-thalassemia, its primary endpoint. Additionally, the study met all the key secondary endpoints, it says.

Thalassemia is described as a rare inherited blood disorder caused by genetic mutations that lead to reduced production of healthy hemoglobin, compromising red blood cell development, health and survival, and resulting in chronic anemia.

Regarding safety, two of 301 patients (0.66%) on mitapivat experienced hepatocellular injury-related adverse events within the first six months after treatment which resulted in treatment discontinuation, according to Agios. “Liver tests improved following discontinuation of mitapivat,” it says. Based on these events, Agios says it included in its FDA submission information about “hepatocellular injury as an important potential risk of mitapivat in patients with thalassemia and proposed monthly monitoring of liver tests for the first six months of treatment with mitapivat.”