Logo

Long-term safety and efficacy of canakinumab in cryopyrin-associated periodic syndrome: results from an open-label, phase III pivotal study in Japanese patients

1, 2, 3, 4, 5, 6, 7, 8

  1. Laboratory of Paediatric Research, Medical Research Institute of Tokyo Medical School, Japan. yokotashumpei@gmail.com
  2. Laboratory of Paediatric Research, Medical Research Institute of Tokyo Medical School, Japan.
  3. Department of Paediatrics, Kyoto University, Kyoto, Japan.
  4. Department of Paediatrics, Kyushu University, Fukuoka, Japan.
  5. Novartis Pharmaceutical Corporation, East Hanover, NJ, USA.
  6. Novartis Pharma AG, Basel, Switzerland.
  7. Department of Paediatrics, Kyoto University, Kyoto, Japan.
  8. Fukuoka Children’s Hospital, Fukuoka, Japan.

CER9166 Submission on line
2017 Vol.35, N°6 ,Suppl.108 - PI 0019, PF 0026
Full Papers

Free to view (click on article PDF icon to read the article)

Rheumatology Article

 

Abstract

OBJECTIVES:
To assess the long-term safety and efficacy of canakinumab in Japanese patients with cryopyrin-associated periodic syndrome (CAPS).
METHODS:
In this open-label phase 3 study, Japanese patients aged ≥2 years with CAPS received canakinumab 2-8 mg/kg subcutaneously every 8 weeks. The duration of the core treatment phase was 24 weeks followed by 22 months extension phase. The primary objective was the proportion of patients free of clinical and serologic relapse at week 24.
RESULTS:
The study enrolled 19 Japanese patients (median age, 14 years; range, 2-48 years) with CAPS [MWS, 7 (36.8%); NOMID, 12 (63.2%)] for a median of 109 weeks. Fifteen patients (79%) achieved a complete response by day 15, 18 (94.7%) by week 24 and all by week 48. At the end of the study, 18 (95%) were free from relapse and 11 (57.9%) were assessed as having no disease activity by the PGA. Thirteen (68%) patients (MWS, 4; NOMID, 9) had their canakinumab dose increased during the trial. All patients experienced at least one adverse event (AE), the most common being infections (100%) and 5 (26.3%) reported serious AEs. No deaths were reported and the only patient who discontinued the study early withdrew consent.
CONCLUSIONS:
Regular canakinumab treatment every 8 weeks at dose levels from 2-8 mg/kg, based on the clinical need, represents a successful strategy to induce rapid and complete response while maintain long-term disease control in Japanese patients with CAPS. The safety profile of canakinumab was consistent with that observed from previous studies.

PMID: 27974104 [PubMed]

Received: 04/12/2015 - Accepted : 22/07/2016 - In Press: 14/12/2016 - Published: 27/11/2017