Clinical requirements for medicines intended for the treatment of thalassaemia - Scientific guideline

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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