The approval is based on results from the ENVISION I Phase 3 study, which showed biologic use linked to clinical remission and maintenance in patients.
The US Food and Drug Administration (FDA) has approved adalimumab (HUMIRA) for treatment in pediatric patients with moderate to severe active ulcerative colitis.
Granted to AbbVie, the approval makes the subcutaneous biologic the first and only of its kinds to treat patients ≥5 years of age with the condition.
"Ulcerative colitis can have a profound effect on children and for too long, treatment options for pediatric patients have been limited," said Brandee Pappalardo, vice president and head of US immunology medical affairs, AbbVie, in a statement.
"This approval provides the first and only subcutaneous biologic for pediatric patients with ulcerative colitis that can be administered at home. This new indication for HUMIRA demonstrates AbbVie's commitment to patients with inflammatory bowel diseases and reinforces our goal of reducing the burden of this disease for patients,” she continued.
The ENVISION I Phase 3 Study
The indication was based on findings from a randomized, double-blind, multicenter, trial that showed that adalimumab induced and maintained clinical remission in patients.
More specifically, the biologic achieved the co-primary endpoints of clinical remission—measured according to the Partial Mayo Score—at week 8 and clinical remission at week 52—per Full Mayo Score.
Clinical remission was defined as a Partial Mayo Score or as a Full Mayo Score ≤2 as well as no individual sub-score >1.
At week 8, these results were reached by 60% of those who received 2.4 mg/kg (maximum of 160 mg) at week 0, 2.4 mg/kg (maximum of 160 mg) at week 1, 1.2 mg/kg (maximum of 80 mg) at week 2, and 0.6 mg/kg (maximum of 40 mg) at weeks 4 and 6.
Among those who received the same dosing schedule excluding dosing at week 1 (or the lower dosing group), 43% achieved clinical remission.
At week 52, 45% of the week 8 responders in the higher dosing group maintained clinical remission—versus 33% of week 8 responders in lower dosing group.
No new safety signals were identified during the course of the study.
However, the investigators noted that 22.6% of patients experienced a serious adverse event. The most frequently reported treatment-emergent adverse events during induction and maintenance were headache and worsening of ulcerative colitis.
Currently, adalimumab is approved in the US for use in 11 indications, which includes 5 approvals in pediatric populations.