The Food and Drug Administration (FDA) has approved Stelara (ustekinumab) injection for the treatment of moderately to severely active ulcerative colitis in pediatric patients aged 2 years and older, the agency announced Aug. 28.
The expansion follows the April 15, 2026, approval of ustekinumab for moderately to severely active Crohn’s disease in pediatric patients aged 2 years and older. With both pediatric indications in place, Stelara is the first approved therapeutic monoclonal antibody that does not target human tumor necrosis factor-alpha (TNF) for the two main forms of pediatric inflammatory bowel disease, according to the FDA.
What supported the indication
The FDA said the safety and effectiveness of Stelara for moderately to severely active UC in children was supported by evidence from adequate and well-controlled studies in adults with UC, pharmacokinetic and safety data from pediatric patients with Crohn’s disease aged 2 to 17 years, and additional safety, efficacy, and pharmacokinetic data from a 52-week study in 112 pediatric patients with UC aged 3 to 17 years.
That 52-week study is the phase 3 UNIFI Jr trial (NCT04630028). According to results published in the Journal of Crohn’s and Colitis, 112 patients with moderately to severely active UC received a single open-label intravenous induction dose of ustekinumab, and 109 were randomized at week 8 to blinded subcutaneous maintenance therapy every 8 weeks or every 12 weeks for 44 weeks. Seventy-nine patients achieved clinical response at week 8.
Among those 79 induction responders, 32 (40.5%) were in clinical remission at week 52, 52 (65.8%) achieved symptomatic remission, 32 (40.5%) achieved endoscopic improvement, and 32 (40.5%) were corticosteroid-free for at least 90 days, investigators reported. Week-52 remission was higher among patients who had not previously failed a biologic than among those who had, 47.2% vs. 26.9%, a subgroup comparison in which the confidence intervals overlapped.
Safety and designation
The most common adverse reactions in pediatric patients with UC receiving Stelara are nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhea, sinusitis, fatigue, and nausea, according to the FDA. In UNIFI Jr, serious adverse events occurred in 6.4% of patients (7 of 109) during maintenance therapy, most commonly gastrointestinal disorders.
Stelara received orphan drug designation for the indication. Approval was granted to Janssen Biotech.
Ustekinumab in adult UC
Ustekinumab targets the p40 subunit shared by interleukin-12 and interleukin-23 and has been approved for adults with moderately to severely active UC since 2019.
AGA’s living guideline on the pharmacological management of moderate-to-severe ulcerative colitis addresses adult outpatients and does not make recommendations for pediatric patients. For adults previously exposed to one or more advanced therapies, particularly TNF antagonists, the guideline groups ustekinumab among the higher-efficacy medications; for adults naive to advanced therapies, it groups ustekinumab among the intermediate-efficacy medications. Guideline panel members reviewed new UC evidence in July 2025 and in March 2026 and issued no new or amended recommendations.
Other pediatric options under study
The approval adds to a small set of advanced therapies labeled for children with UC. In December 2025, Jess L. Kaplan, MD, of the Division of Pediatric Gastroenterology and Nutrition at Mass General for Children, Boston, told GI & Hepatology News that anti-TNF agents were at that time the only FDA-approved biologic or advanced therapy for children with moderate to severe UC. Dr. Kaplan was first author on a phase 2 trial of the interleukin-23 inhibitor mirikizumab in children with UC.
GI & Hepatology News reported in January 2026 on real-world outcomes with off-label upadacitinib in 48 patients younger than 18 years with Crohn’s disease or UC, presented at the 2026 Crohn’s & Colitis Congress.