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FDA approves Siklos for children with sickle cell anemia
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The FDA approved hydroxyurea to reduce crises and the need for blood transfusions among children aged 2 years or older with sickle cell anemia who have recurrent moderate to severe painful crises.
Hydroxyurea (Siklos, Addmedica) — which helps reduce the production of sickle hemoglobin — is approved for the treatment of adults with severe symptoms of sickle cell disease. This is the first FDA approval of hydroxyurea for use in children.
The FDA based the approval on results from the open-label single-arm ESCORT study, which evaluated the safety and efficacy of hydroxyurea among 405 children aged 2 to 18 years with sickle cell anemia. The study included an efficacy cohort of 141 children who had not previously been treated with hydroxyurea.
After 12 months of hydroxyurea treatment (20 mg/kg once daily), researchers observed a decrease in the percentage of patients with at least one vaso-occlusive episode, one episode of acute chest syndrome, one hospitalization due to sickle cell disease or one blood transfusion.
The most common adverse reactions associated with hydroxyurea included infection and neutropenia.
The FDA granted priority review and orphan drug designation to this application.