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Phase 3 study of mitapivat to reduce transfusion burden in sickle cell disease
2026-526778-18-00Phase 3Authorised
- Sponsor
- Agios Pharmaceuticals
- Start
- Sep 2026
- Modality
- Small Molecules
- Countries
- 15
01
Overview
Phase 3, randomised, double-blind trial in sickle cell disease (SCD) comparing Mitapivat with Placebo (2 arms). Primary endpoint: Transfusion-free response. Planned start Sep 2026; status: authorised.
02
Drugs
mitapivat
03
Conditions
sickle cell disease (SCD)
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