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FDA Approves Hydroxyurea Tablets for Pediatric Sickle Cell Anemia

The U.S. Food and Drug Administration (FDA) has approved hydroxycarbamide/hydroxyurea tablets (Siklos) in pediatric patients suffering from sickle cell anemia, ages two years and older, France-based Addmedica has announced.

The therapy is the first and only hydroxyurea-based treatment for the indication.

The approval was based on positive safety and efficacy data collected in the Escort-HU study (NCT02516579). It had previously been granted orphan drug designation and priority review.

In the trial, the antimetabolite was used in current practices for the prevention of symptomatic vaso-occlusive complications in several hundred pediatric and adult patients with sickle cell anemia. After 12 months of treatment, the drug demonstrated the ability to increase fetal hemoglobin in all patients, while decreasing the percentage of patients who experienced at least one vaso-occlusive episode, one episode of acute chest syndrome, one hospitalization due to sickle cell disease, or one blood transfusion.