Sobi announces FDA approval of Doptelet for pediatric thrombocytopenia patients
Sobi North America announced that the U.S. Food and Drug Administration approved Doptelet for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia who have had an insufficient response to a prior therapy. The approval also includes a new formulation, Doptelet Sprinkle oral granules, for use in children ages one to less than six years. Doptelet Sprinkle is the approved dosage form for patients aged one to less than six years, while Doptelet tablet is the approved dosage form for patients aged six and up. The pediatric approval is based on results from AVA-PED-301, a global, randomized, phase 3 study evaluating the efficacy, safety, and pharmacokinetics of Doptelet in the treatment of pediatric subjects with ITP. The study showed that 27.8% of Doptelet patients achieved the primary endpoint of durable platelet response versus 0% of placebo patients in the absence of rescue medication. Also, 81.5% of Doptelet patients achieved the alternative primary endpoint of platelet response versus no patients in the placebo group. Doptelet was generally well-tolerated. In pediatric patients with persistent or chronic immune thrombocytopenia, the most common adverse reactions were viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.
