Clinical requirements for medicines intended for the treatment of thalassaemia

This guideline aims at providing guidance on general principles of the clinical development programme of medicinal products for the treatment of α and β-thalassaemia. This guideline focuses on the confirmatory phase 3 trials investigating safety and efficacy and serving as the main basis for benefit risk assessment of these medicines.

Keywords: α-thalassaemia, β-thalassaemia, transfusion-dependent, non-transfusiondependent, iron overload

Current version

Draft guideline on the clinical requirements for medicines intended for the treatment of thalassaemia

Draft: consultation open Consultation dates: 24/09/2026 to 31/01/2027 Reference Number: EMA/CHMP/HAEMWP/57392/2025 Summary:

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