Efficacy and safety of canakinumab in systemic juvenile idiopathic arthritis: 48-week results from an open-label phase III study in Japanese patients

Abstract

Objectives

To assess the efficacy and safety of canakinumab in Japanese patients with systemic juvenile idiopathic arthritis (sJIA).

Methods

This was an open-label, single-arm active treatment study. sJIA patients, aged ≥2 to <20 years, were administered canakinumab 4 mg/kg every 4 weeks for ≤48 weeks. The co-primary endpoints were the proportion of patients who achieved an adapted American College of Rheumatology pediatric (ACR pedi) 30 criteria at week 8, and the proportion of patients who successfully tapered corticosteroids at week 28. Herein, the efficacy and safety results up to 48 weeks are reported.

Results

Of the 19 patients enrolled, 15 (78.9%) had previously used tocilizumab. All patients achieved ACR pedi 30 at week 8 and 73.7% (14/19) successfully tapered corticosteroids at week 28. At week 48, ACR pedi 50/70/90/100 responses were achieved by 100.0%/100.0%/87.5%/68.8% of patients. The most common adverse events (AEs) were infections (271.6 patient-years), 42.1% (8/19) patients had serious AEs. Two potential cases of macrophage activation syndrome were identified. No deaths were reported.

Conclusion

Canakinumab was efficacious in Japanese patients with sJIA and was associated with substantial corticosteroid dose reduction in the majority of patients. The safety profile of canakinumab was consistent with that observed from previous studies.

ClinicalTrials.gov (identifier

NCT02396212).

Keywords:

• Canakinumab
• corticosteroid tapering
• interleukin-1β
• macrophage activation syndrome
• systemic juvenile idiopathic arthritis

Acknowledgements

The authors thank Amit Agarwal, Novartis Healthcare Pvt. Ltd, India for medical writing support.

Conflict of interest

ST: Consultancy fee from Bristol-Myers Squibb. Speaking fees, and honoraria from Chugai, Abbvie, Ono, Takeda, Tanabe-Mitsubishi, Sanofi, Glaxo-Smith-Kline.

NI: Consultancy fee from Tanabe-Mitsubishi Pharm, GlaxoSmithKline. Speaking fees and honoraria from Mitsubishi Tanabe Pharma Corporation, Novartis Pharma K.K., Janssen Pharmaceutical K.K., AbbVie GK.

MS: Speaking fees and honoraria from Novartis Pharma K.K.

NS, TK: Employees of Novartis Pharma K.K.

The rest of the authors declare that they have no competing interests.

Author contributions

NS and TK conceived and planned the study. KN, RH, HU, ST, NI, TI, MS, MT, and SY substantially contributed to analysis and interpretation of data. KN took the lead in writing the manuscript. All authors provided critical feedback and helped shape the research, analysis, and manuscript.

Additional information

Funding

This study was funded by Novartis Pharma AG, Basel, Switzerland.