FDA Approves Hydroxyurea for Treatment of Paediatric Patients with Sickle Cell Anaemia

Approval is based on data from the European Sickle Cell Disease Cohort study

On 21 December 2017, the US Food and Drug Administration (FDA) granted regular approval to hydroxyurea (Siklos, Addmedica) to reduce the frequency of painful crises and the need for blood transfusions in paediatric patients from 2 years of age and older with sickle cell anaemia with recurrent moderate to severe painful crises.

This is the first FDA approval of hydroxyurea for use in paediatric patients with sickle cell disease.

Approval was based on data from an open-label single-arm trial, the European Sickle Cell Disease Cohort study (ESCORT HU, NCT02516579), of 405 paediatric patients with sickle cell disease from 2-18 years of age, of which 141 had not been previously treated with hydroxyurea prior to enrolment. Among this paediatric population analysable for efficacy (n = 141), Siklos use resulted in an increase in haemoglobin F. The percentage of patients with at least one vaso-occlusive episode, one episode of acute chest syndrome, one hospitalisation due to sickle cell disease or one blood transfusion decreased after 12 months of hydroxyurea treatment.

Most common adverse reactions to Siklos (incidence >10%) include infections and neutropenia.

The recommended initial dose of hydroxyurea is 20 mg/kg once daily.

Full prescribing information is available here

FDA granted priority review and Orphan Drug designation to this application.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System.