SAT0522 Canakinumab Pharmacokinetics in Patients Younger Than 2 Years Old with Cryopyrin Associated Periodic Syndromes

Background Canakinumab (CAN) has demonstrated efficacy in patients with Cryopyrin-Associated Periodic Syndromes (CAPS) ≥2 yrs of age 1 . However, no information on the pharmacokinetics (PK) of CAN in patients 2 yrs of age is available. We present preliminary PK data from a phase III study in this patient population. Objectives Primary objective was to assess the efficacy of CAN with respect to the treatment response in CAPS patients ≤4 yrs of age. A secondary endpoint was to evaluate PK and pharmacodynamics (PD) profiling of CAN. Methods CAN-naive patients with confirmed CAPS aged 44 days to 4 yrs received open-label CAN dosed 2-12 mg/kg every 4 or 8 weeks for 56 weeks. For NOMID patients, an initial dose of 4 mg/kg was administered. Patients who did not achieve complete response following CAN injection, or experienced a flare before the next planned administration, were eligible for dose up-titration with possible maintenance and step-wise up-titration regimens of 4, 6, 8, 10, or 12 mg/kg s.c. Results Seventeen patients, 6 patients 2 yrs old who received CAN doses ranging from 2–10 mg/kg. The safety profile for patients Conclusions Canakinumab is a highly effective treatment for patients with CAPS aged as young as 44 days old. The preliminary PK results demonstrated that dose-normalized canakinumab exposure in patients 2 yrs supporting the utilization of weight-based dosing in the CAPS infantile population. References Lachmann H, et al. N Engl J Med. 2009;60:2416-25. Disclosure of Interest J. Kalabus Employee of: Novartis, Y. Uziel Consultant for: Novartis, Speakers bureau: Novartis, P. Brogan Grant/research support from: Institutional grant support to undertake the study described in this abstract, Consultant for: SOBI and Roche, M. Hofer Consultant for: Novartis, J. Kuemmerle-Deschner Grant/research support from: Novartis, Consultant for: Novartis, B. Lauwerys: None declared, A. Speziale Employee of: Novartis, R. Laxer Grant/research support from: Database funding from Novartis, Consultant for: Participant in Ad Board for Novartis, H. Lachmann Consultant for: Novartis, Speakers bureau: Novartis, H. Sun Employee of: Novartis, K. Abrams Shareholder of: Novartis, Employee of: Novartis, K. Leon Employee of: Novartis, G. Junge Employee of: Novartis