The Food and Drug Administration (FDA) has expanded the approval of Humira® (adalimumab; AbbVie) to include the treatment of moderately to severely active ulcerative colitis in patients 5 years of age and older.
The pediatric approval was based on data from the multicenter, randomized, double-blind phase 3 ENVISION I study (ClinicalTrials.gov: NCT04131504), which assessed the efficacy and safety of Humira in patients 5 to 17 years of age with moderately to severely active ulcerative colitis (defined as a Mayo Score of 6 to 12 with endoscopy subscore of 2 to 3 points, confirmed by centrally read endoscopy), who had an inadequate response or intolerance to therapy with corticosteroids and/or an immunomodulator.
Patients received 2.4mg/kg (maximum 160mg) at week 0, followed by 1.2mg/kg (maximum 80mg) at week 2, and 0.6mg/kg (maximum 40mg) at weeks 4 and 6. The higher dosage group received an additional dosage of 2.4mg/kg (maximum 160mg) at week 1.
At week 8, patients who demonstrated clinical response per Partial Mayo Score (a subset of the Mayo Score with no endoscopic component and defined as a decrease in Partial Mayo Score ≥2 points and ≥30% from baseline) were randomly assigned to receive double-blind treatment with Humira 0.6mg/kg (maximum 40mg) every other week (lower dosage group), 0.6mg/kg every week (higher dosage group), or placebo.
The co-primary end points were clinical remission per Partial Mayo Score at week 8, and clinical remission per the Mayo Score (defined as Mayo Score ≤2 and no individual subscore >1) at week 52 in patients who achieved clinical response per Partial Mayo Score at week 8.
Results showed that at week 8, 60% (n=28/47; 95% CI, 44-74) of patients treated with the higher dosage of Humira and 43% (n=13/30; 95% CI, 25-63) of patients in the lower dosage group achieved clinical remission per Partial Mayo Score. At week 52, among Partial Mayo Score responders, 45% (n=14/31; 95% CI, 27-64) of patients treated with the higher dosage of Humira, 29% (n=9/31; 95% CI, 14-48) of patients treated with the lower dosage, and 33% (n=4/12; 95% CI, 10-65) of those who received placebo achieved clinical remission per the Mayo Score.
The most frequently reported treatment-emergent adverse events (incidence greater than or equal to 5%) were headache and worsening of ulcerative colitis.
“This approval provides the first and only subcutaneous biologic for pediatric patients with ulcerative colitis that can be administered at home,” said Brandee Pappalardo, vice president and head of US immunology medical affairs, AbbVie.
Humira® (adalimumab) receives FDA approval to treat pediatric patients living with moderately to severely active ulcerative colitis. [press release]. North Chicago, IL: AbbVie; February 24, 2021.