Regulatory & Policy

FDA expands Stelara into pediatric ulcerative colitis with non-TNF mechanism

FDA expands Stelara into pediatric ulcerative colitis with non-TNF mechanism

Stelara (ustekinumab), the anti-IL-12/23 monoclonal antibody marketed by Janssen Biotech, has received US FDA approval for moderately to severely active ulcerative colitis (UC) in pediatric patients aged 2 years and older — making Stelara the first non-TNF monoclonal antibody approved in the US for both pediatric ulcerative colitis and pediatric Crohn’s disease. Janssen had secured a parallel pediatric approval for Crohn's disease (CD) in April 2026, completing coverage across the two principal IBD diagnoses in children.

The FDA granted Stelara Orphan Drug Designation for the pediatric UC indication. The approval was supported by adult UC efficacy data, pharmacokinetic and safety data from pediatric CD patients aged 2 to 17, and a dedicated 52-week study enrolling 112 pediatric UC patients aged 3 to 17 years. The most common adverse reactions reported in pediatric UC patients included nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhea, sinusitis, fatigue, and nausea.

Ustekinumab binds the p40 subunit shared by IL-12 and IL-23, blocking their interaction with the IL-12Rβ1 receptor on T cells and natural killer cells. This suppresses Th1- and Th17-driven mucosal inflammation — a mechanism distinct from the TNFα blockade used by anti-TNF biologics.

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The non-TNF distinction sets these agents apart from anti-TNF agents, which remain the backbone of biologic therapy in pediatric IBD. Johnson & Johnson's Simponi (golimumab), an anti-TNFα antibody from the same corporate family, received FDA approval for pediatric UC in patients weighing at least 15 kg in October 2025. Takeda's vedolizumab (Entyvio), a gut-selective anti-integrin antibody, has an sBLA under FDA review for pediatric UC and CD with a PDUFA target date in Q1 2027, and would offer a mechanistically distinct alternative if approved. AbbVie's risankizumab (Skyrizi), a selective IL-23 p19 inhibitor approved for adult UC and CD, has not yet secured pediatric IBD labeling.

Stelara's adult UC franchise also faces growing biosimilar competition. In the broader UC pipeline, France-based Abivax's obefazimod has reported Phase III maintenance data in adult patients and is targeting an NDA filing in Q4 2026.


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