Efficacy and safety of febuxostat in Japanese paediatric patients with hyperuricaemia including gout: Phase 2, single arm, open‒label, multicentre studies.

Objectives: Urate-lowering efficacy and safety of febuxostat was evaluated in paediatric patients with hyperuricaemia including gout.

Methods: A Phase 2 study of febuxostat in paediatric patients aged 6-18 years with hyperuricaemia including gout was conducted. We evaluated the proportion of patients achieving serum uric acid (sUA) level ≤6.0 mg/dl at Week 26, and long-term safety and efficacy at Week 52. We also considered efficacy stratified by renal function.

Results: Thirty patients (10 at <40 kg and 20 at ≥40 kg) were enrolled. Twenty-four were males, 29 had asymptomatic hyperuricaemia, and 1 had gout. Age was 8 to 18 years. Of these, 63.3% (95% confidence interval 43.9-80.1%) achieved a sUA level of ≤6.0 mg/dl at Week 26. sUA level (mean ± standard deviation) was 5.55 ± 0.87 mg/dl, reduced from 9.01 ± 1.23 mg/dl at baseline. Febuxostat efficacy appeared similar for mild to moderate renal dysfunction and with normal renal function. There were no major safety issues.

Conclusions: In paediatric patients with hyperuricaemia including gout, febuxostat showed long-term, well-controlled urate-lowering efficacy with no major safety issues. Findings suggest that no dose adjustment is required for paediatric patients with mild to moderate renal dysfunction.