Safety and effectiveness of canakinumab in Japanese patients with cryopyrin-associated periodic syndrome: final results from a post-marketing surveillance in Japan

ObjectivesTo assess the real-world safety and effectiveness of canakinumab, a monoclonal anti-interleukin-1β antibody, in patients with cryopyrin-associated periodic syndrome (CAPS) in a real-world setting in Japan. MethodsAll patients with CAPS who received canakinumab treatment after drug approval in Japan were registered in a post-marketing all-patient surveillance with 2-year observation period and a follow-up period of up to 5 years. ResultsOf 93 patients in the safety analysis set, the proportion of patients with any adverse drug reactions (ADRs) and any serious ADRs was 33.33% and 4.30%, respectively. The most common ADRs were infections and infestations (21.51%). Of 70 new and 18 continuing patients (roll-over from a previous clinical trial) in the effectiveness analysis set, the proportion of responders among new and continuing patients who achieved both clinical and serological remission, was 78.57% and 83.33%, respectively at Week 24. Most responders remained without relapse after the remission until Week 104 (98.11% of new and 100% of continuing patients). Clinical symptoms related to auditory, joint, visual, renal, and central nervous system maintained or improved in majority of patients. ConclusionsCanakinumab was well-tolerated and effective during long-term treatment for patients with CAPS in the real-world setting.