Mitapivat
Mitapivat is an oral pyruvate kinase activator developed by Agios Pharmaceuticals, sold as PYRUKYND and AQVESME. The next event is the FDA decision on Mitapivat (supplemental application), on Nov 1, 2026 (source).
- Also known as
- PYRUKYND, AQVESME
- Company
- Agios Pharmaceuticals (AGIO) · Developer
- How it works
- Pyruvate kinase activator
- Approved
- PYRUKYND for pyruvate kinase deficiency: United States, United Kingdom
- PYRUKYND for thalassemia: European Union, United Arab Emirates
- AQVESME for thalassemia: United States
Development by disease
| Pyruvate kinase deficiency | Approved |
| Thalassemia | Approved |
| Sickle cell disease | Filed |
Upcoming events
Firm dates · a specific day set by the company or regulator
Next 30 days
- Nov 1in 19 daysMitapivat · FDA decision (supplemental application) · Sickle cell disease
Past results and milestones
- Jul 30, 2026The REIGNITE confirmatory Phase 3 trial was initiated to evaluate mitapivat clinical benefit on transfusion burden in sickle cell disease. Source
- May 1, 2026European Commission granted marketing authorization for PYRUKYND (mitapivat) in May 2026 for treatment of anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia with orphan medicinal product designation. Source
- Apr 29, 2026Pre-sNDA meeting with FDA was completed in Q1 2026. Agios confirmed pursuit of the U.S. accelerated approval pathway for mitapivat in sickle cell disease and planned the sNDA submission for Q2 2026. Source
- Dec 23, 2025FDA approved AQVESME (mitapivat) in December 2025 for treatment of anemia in adults with alpha- or beta-thalassemia. Source
- Nov 1, 2025RISE UP Phase 3 topline results reported in November 2025 for mitapivat in sickle cell disease, supporting planned regulatory submission. Source
- Feb 13, 2025Positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1–18 years with pyruvate kinase deficiency who are not regularly transfused were announced, making it the first oral therapy to demonstrate efficacy in this pediatric population. Source