FDA Pushes Back Agios’ Pyrukynd Thalassemia Decision to December

The FDA has delayed its decision on Agios Pharmaceuticals' supplemental New Drug Application (sNDA) for Pyrukynd (mitapivat) in thalassemia by three months, setting a new action date of December 7, 20251235.

The delay results from Agios submitting a proposed Risk Evaluation and Mitigation Strategy (REMS) to address the risk of hepatocellular injury (liver damage) associated with Pyrukynd123.

This extension is procedural, not based on new safety or efficacy data, but rather to allow the FDA sufficient time to evaluate the REMS submission123.

Pyrukynd is already approved for hemolytic anemia in adults with pyruvate kinase deficiency in the US, EU, and Great Britain, and was recently approved for thalassemia in Saudi Arabia13.

The current sNDA is supported by data from the ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia3.

Sources:

1. https://www.ainvest.com/news/fda-delays-decision-agios-pharmaceuticals-pyrukynd-expanded-blood-disorder-treatment-2509/

2. https://www.ainvest.com/news/agios-pharmaceuticals-fda-extends-pdufa-goal-date-pyrukynd-thalassemia-december-7-2025-2509/

3. https://www.benzinga.com/news/fda/25/09/47500813/fda-delays-decision-on-agios-pharmaceuticals-blood-disorder-drug-for-expanded-use

5. https://www.zacks.com/stock/news/2747555/agio-stock-falls-as-fda-delays-decision-on-pyrukynd-for-thalassemia