New pediatric sickle cell anemia therapy approved in the USA

15 March 2018

Paris-based ADDMEDICA has announced that the US Food and Drug Administration has approved its sickle cell anemia therapy Siklos (hydroxyurea tablets) for children aged two and over, under a priority review and orphan drug designation.

The therapy offers dosing options to facilitate adjustment based on the patient's weight to optimize the efficacy/tolerance ratio for children.

The approval was granted based on efficacy and safety data collected in a cohort study, Escort-HU, which included several hundred child and adult patients treated with Siklos in Europe.

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