Sickle Cell Anemia Drug Gains FDA Approval

Treatment with hydroxyurea increased hemoglobin F levels for patients with sickle cell anemia.

The FDA today granted regular approval to hydroxyurea (Siklos) 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, according to a press release.

This was the first approval for hydroxyurea indicated for pediatric patients with sickle cell disease, the FDA said.

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.

Common adverse reactions occurring in less than 10% of hydroxyurea-treated patients included infections and neutropenia.

The FDA previously granted hydroxyurea orphan drug designation, according to the release.

Earlier this year, the FDA approved L-glutamine oral powder (Endari) for the treatment of sickle cell disease in patients 5 years and older, making it the first drug approved in 20 years.

The approval of both therapies will likely make treatment accessible for many patients with sickle cell disease.