FDA Approves Sickle Cell Anemia Drug – APhA submits compounding comments to FDA. – (866) 348-2889
Officials with the FDA have approved hydroxyurea (Siklos) as the first treatment indicated for pediatric patients with sickle cell disease, according to a press release.
Hydroxyurea is indicated to decrease the prevalence of painful crises and the need for transfusions in pediatric patients aged 2 years and older with sickle cell anemia with moderate-to-severe painful crises.
The approval was based on findings from the European Sickle Cell Disease Cohort study, which included 405 patients with sickle cell disease aged 2 to 18 years, of whom 141 were not previously-treated.
The investigators found that treatment with hydroxyurea resulted in increased hemoglobin F levels, according to the release.
The prevalence of patients who experienced at least 1 vaso-occlusive episode, 1 episode of acute chest syndrome, 1 hospitalization, or 1 blood transfusion was reduced after 12 months of treatment, according to the FDA.