Regulatory Decision Summary for Humira

Humira was evaluated in a randomized, double-blind, multicentre study that included 93 pediatric patients ranging from 5 to 17 years of age with moderate to severe ulcerative colitis who had inadequate response or intolerance to conventional therapy. Approximately 20% of the patients were 12 years of age and younger. Patients were to be randomized in a 3:2 ratio at baseline to receive either a high or standard dose Humira induction therapy followed at week 8 by randomization to one of two Humira maintenance doses until 52 weeks.

The co-primary efficacy endpoints of the study were clinical remission per Partial Mayo Score (PMS-defined as score ≤ 2 and no individual subscore > 1) at Week 8, and clinical remission per Full Mayo Score (FMS-defined as a Mayo Score ≤ 2 and no individual subscore > 1; with endoscopy) at Week 52 in patients who achieved clinical response per PMS at Week 8 following blinded therapy. Primary efficacy analysis revealed a favourable efficacious response for the primary endpoints. At week 8, patients in both induction groups achieved remission as measured by PMS. A greater proportion of subjects that were responders per PMS at week 8, achieved clinical remission as measured by Mayo score at week 52 compared to external placebo. The recommended fixed-dose dosing regimen included in the product monograph is expected to be reflective of results from the high induction and maintenance groups.

The most frequently reported adverse events were related to infections and infestations (53.8%), gastrointestinal disorders (50.5%) and nervous system disorders (28.0%). Colitis ulcerative, headache, anemia, upper respiratory tract infection, nasopharyngitis, and pharyngitis were the most frequently reported treatment emergent adverse events. No deaths were reported in the studies. Overall, the safety profile of Humira remained consistent with prior knowledge and no new safety signals in the pediatric population were detected.

The exposure of pediatric patients with ulcerative colitis to Humira is limited. In addition to an extended history of use in other pediatric indications with favourable safety and efficacy profiles, the evaluation of the longer-term safety and efficacy of Humira in pediatric patients with ulcerative colitis remains ongoing in an open-label extension study. Data from this study will be provided to Health Canada upon study completion.

Humira is expected to provide an additional treatment option for pediatric patients with moderate to severe ulcerative colitis who experience an inadequate response or intolerance to conventional therapy. Overall, the benefit/risk profile of Humira is considered to be favourable for pediatric patients with ulcerative colitis.