Families managing a child's ulcerative colitis gained a treatment option last week that did not exist for them before. The Food and Drug Administration approved Stelara for moderately to severely active ulcerative colitis in patients aged 2 years and older on Aug. 28.
Until now, the advanced therapies formally labeled for children with moderate to severe ulcerative colitis all targeted tumor necrosis factor. Not every child responds to that class, and some who do lose response over time. Parents in that position have generally faced off-label prescribing, insurance appeals, or both.
The approval matters most to a specific group. It is the child who has already failed an anti-TNF drug, whose family has been fighting a prior authorization denial, and for whom the next conversation would otherwise have started with the words not approved for children. A labeled indication changes the terms of that conversation with an insurer, though it does not guarantee coverage.
The Evidence Behind the Label
The FDA said the approval rested on three sources of evidence: adequate and well-controlled studies in adults with ulcerative colitis, pharmacokinetic and safety data from children with Crohn's disease aged 2 to 17, and a 52-week study in 112 pediatric patients with ulcerative colitis aged 3 to 17.
That last study is the Phase 3 UNIFI Jr trial, registered as NCT04630028. According to results published in the Journal of Crohn's and Colitis and summarized by GI and Hepatology News, 112 patients received a single open-label intravenous induction dose and 109 were randomized at week 8 to blinded subcutaneous maintenance dosing every 8 or 12 weeks for 44 weeks. Seventy-nine achieved a clinical response at week 8.
Among those 79 responders, 32 were in clinical remission at week 52, 52 achieved symptomatic remission, 32 achieved endoscopic improvement, and 32 were free of corticosteroids for at least 90 days. Serious adverse events occurred in 6.4 percent of patients during maintenance, seven of 109, most commonly gastrointestinal disorders.
Two limitations belong up front rather than buried. The trial had no placebo arm, and the pediatric approval leans substantially on extrapolation from adult data. And the week-52 remission figures describe only the subgroup that responded by week 8, not the full enrolled population. Remission was numerically higher among children who had not previously failed a biologic, at 47.2 percent versus 26.9 percent, but the confidence intervals for that comparison overlapped, which means the difference was not statistically established.
Where This Sits Among Existing Options
Stelara targets the p40 subunit shared by interleukin-12 and interleukin-23, and it has been approved for adults with moderately to severely active ulcerative colitis since 2019. The FDA approved it for pediatric Crohn's disease on April 15. With both indications in place, the agency says it becomes the first approved therapeutic monoclonal antibody that does not target TNF for the two main forms of pediatric inflammatory bowel disease.
Independent context is worth noting. Jess L. Kaplan, a pediatric gastroenterologist at Mass General for Children, told GI and Hepatology News in December 2025 that anti-TNF agents were at that point the only FDA-approved advanced therapy for children with moderate to severe ulcerative colitis. Kaplan was first author on a Phase 2 trial of the interleukin-23 inhibitor mirikizumab in the same population, and other candidates remain under study.
The American Gastroenterological Association's living guideline on ulcerative colitis addresses adult outpatients and makes no pediatric recommendations. For adults previously exposed to advanced therapies, it groups ustekinumab among higher-efficacy medications. For adults new to advanced therapies, it groups the drug among intermediate-efficacy options. Panel members reviewed new evidence in July 2025 and again in March 2026 and issued no new or amended recommendations.
Approval was granted to Janssen Biotech, and the indication received orphan drug designation. Johnson and Johnson develops and markets the drug and funded the trials supporting the application.
Cost and Access Questions Families Should Raise
A labeled pediatric indication typically strengthens a coverage argument, but it does not eliminate prior authorization, step therapy requirements, or specialty pharmacy hurdles. Families should ask the gastroenterology team three concrete questions: whether the plan requires a documented anti-TNF failure first, whether induction dosing is billed as a medical or pharmacy benefit, and what the appeal timeline looks like if the initial request is denied.
Biosimilar competition has entered this market. The FDA has approved multiple ustekinumab biosimilars, and several launched in the United States beginning in early 2025. Whether a given plan prefers the reference product or a biosimilar affects out-of-pocket cost, and families should ask which product the plan lists as preferred before the first infusion is scheduled. A biosimilar approval also does not automatically carry every indication the reference product holds, which is worth confirming for a young child.
Manufacturer patient assistance and copay support programs exist for biologics in this class. Hospital financial counselors and specialty pharmacy teams can usually identify which programs a family qualifies for, and Medicaid coverage rules differ meaningfully by state.
The Reasonable Expectation for Families
Nothing about this approval requires a change to a child's current treatment. Children doing well on an existing therapy have no reason to switch, and no one should stop or alter a prescribed medication without the treating gastroenterologist.
The most common adverse reactions reported in pediatric ulcerative colitis patients were nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhea, sinusitis, fatigue, and nausea. Drugs that suppress immune signaling carry infection risk, and screening before starting is standard.
Longer-term pediatric safety data will accumulate through post-approval surveillance and registries. Families weighing a switch should ask what the plan is if the drug does not work by week 8, since the trial design suggests that is when response is assessed, and should ask how growth and nutrition will be monitored alongside disease activity.
Key Questions Answered
What did the FDA approve? Stelara injection for moderately to severely active ulcerative colitis in patients aged 2 years and older, approved Aug. 28.
Why is this considered a first? With the April approval for pediatric Crohn's disease already in place, the FDA says Stelara becomes the first approved therapeutic monoclonal antibody that does not target TNF for both main forms of pediatric inflammatory bowel disease.
How strong is the pediatric evidence? The 52-week trial enrolled 112 children aged 3 to 17 and had no placebo arm. Approval also relied on adult efficacy data and pediatric Crohn's disease safety data. Of 79 week-8 responders, 32 were in clinical remission at week 52.
Does approval mean insurance will cover it? Not automatically. A labeled indication generally strengthens a coverage request, but plans may still require prior authorization or a documented failure of another therapy first.
What are the most common side effects? Nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhea, sinusitis, fatigue, and nausea, according to the FDA.
Should a child currently doing well switch to this drug? No change is indicated on the basis of an approval alone. Treatment decisions belong with the child's gastroenterologist.
Do biosimilars affect the cost? Possibly. Multiple ustekinumab biosimilars are FDA approved and several have launched, and which product a plan prefers can change out-of-pocket cost. Families should confirm the preferred product before scheduling infusions.
