Target product profile for formulations of hydroxyurea for the management of sickle cell disease in children

Overview

This target product profile (TPP) was developed by the World Health Organization to guide the development, manufacturing and procurement of age-appropriate hydroxyurea formulations for the management of sickle cell disease (SCD) in children and adolescents. SCD is one of the most common inherited disorders worldwide and is associated with substantial morbidity and mortality, particularly in low- and middle-income countries where access to diagnosis, treatment and comprehensive care remains limited. Although hydroxyurea is the main disease-modifying therapy for SCD and is strongly recommended for children and adolescents with sickle cell anaemia, currently available formulations often present challenges for accurate dosing and administration in younger children.

The document describes the WHO-led process used to develop the TPP and presents consensus-based preferred and minimum product characteristics for paediatric hydroxyurea formulations. Key considerations include indication, target population, dosage strength, pharmaceutical form, administration requirements, service delivery level, stability, packaging, cost and regulatory pathways. The TPP emphasizes the need for functionally scored soluble or dispersible tablets that support flexible weight-based dosing, ease of administration and suitability for resource-limited settings. The proposed characteristics are intended to facilitate access to quality-assured formulations and support broader implementation of WHO recommendations for the management of SCD in children and adolescents.