The approval was granted based on efficacy and safety data from the Escort-HU study, which included 495 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.
In regard to safety, adverse events (AEs) occurred less frequently in children (17.3%) than adults (30.8%; P <.01) in the in-label subgroup, with no statistical difference in AEs in the off-label subgroup. In children, infections were more frequent in children than adults (P <.01), which is usually observed, the investigators noted. In total, all other AEs were more frequent in adults.
The study investigators noted that the use of the drug was more frequent off-label in children.
“FDA-approval of Siklos is a major breakthrough in the treatment of American children with sickle cell anemia. European patients have been benefitting from Siklos, the only form of hydroxyurea indicated in pediatric care in the world, since 2007,” François Anger, the president of ADDMEDICA, said in a statement. “This successful outcome can also be attributed to the patients who agreed to participate in the Escort-HU study and the physicians who allowed it to happen. With the new FDA-approval, ADDMEDICA consolidates its leadership in the treatment of sickle cell anemia by continuing to invest in the development and availability of this essential therapeutic product in new countries.”
The treatment was approved for use in Europe in 2007 and will be marketed in the United States by Medunik USA, Inc., according to Éric Gervais, the executive vice-president of Medunik.
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