FDA Approves Hydroxyurea Tablets for Pediatric Patients With Sickle Cell Anemia

The US Food and Drug Administration (FDA) has approved hydroxyurea tablets (Siklos, ADDMEDICA) for the treatment of sickle cell anemia in pediatric patients aged 2 years and older.

Previously, the hydroxyurea tablets were granted priority review and orphan drug designations from the FDA. Sickle cell anemia, by recent estimates, impacts roughly 100,000 Americans.

The approval was granted based on efficacy and safety data from the Escort-HU study, which included 1047 patients with sickle cell anemia (495 children). The mean hydroxycarbamide levels in the pediatric patients at initiation was 18 mg/kg and 17.3 mg/kg.