FDA Approves Ustekinumab Injection for Pediatric Ulcerative Colitis, Expanding IBD Options for Children

The FDA has approved ustekinumab (Stelara; Janssen Biotech) injection for moderately to severely active ulcerative colitis (UC) in pediatric patients aged 2 and older. The approval follows the agency's April 2026 clearance of ustekinumab for pediatric Crohn's disease (CD) in the same age group, making it the first approved therapeutic monoclonal antibody that does not target tumor necrosis factor alpha (TNFα) for either major form of pediatric inflammatory bowel disease (IBD). Ustekinumab received an orphan drug designation for this indication.¹

What Is Ustekinumab?

Ustekinumab is a human IgG1κ monoclonal antibody that targets the shared p40 subunit of interleukin-12 (IL-12) and interleukin-23 (IL-23), 2 cytokines implicated in the chronic mucosal inflammation that drives both CD and UC. By blocking p40, the drug disrupts IL-12/IL-23 signaling through the shared IL-12Rβ1 receptor chain, distinguishing its mechanism from TNFα inhibitors already used across IBD. In pediatric UC, dosing follows the same 2-phase pattern established in CD and adult UC: a single weight-based intravenous induction infusion, followed by subcutaneous maintenance dosing every 8 weeks.^2,3

Clinical Trials and Evidence Supporting Approval

Pediatric UC efficacy and safety were supported by a combination of prior adult UC trial data, pharmacokinetic and safety findings from pediatric CD patients aged 2 to 17, and results from the Phase 3 UNIFI Jr trial (NCT04630028), a 52-week, multicenter study of 112 pediatric patients aged 3 to 17 with moderately to severely active UC who had an inadequate response or intolerance to conventional or biologic therapy. UNIFI Jr used an open-label intravenous induction phase followed by a randomized, double-blind subcutaneous maintenance phase, tracking participants in clinical response from week 8 through week 52.^1,2

The trial builds on the pivotal adult UC program (UC-1 induction, UC-2 maintenance), in which ustekinumab produced significantly higher rates of clinical remission and endoscopic improvement than placebo at both week 8 and week 44. In the pediatric maintenance substudy, adverse events occurred in 81.0% of patients, with infections—nonserious—reported in 57.1%; serious adverse events occurred in 9.5%, and the safety profile was consistent with no new signals identified.^2,3

Clinical Implications

For pharmacists supporting pediatric gastroenterology teams, this approval adds a non-TNF biologic option for children as young as 2 with moderately to severely active UC—a population that often experiences more severe disease than adults and has had limited approved biologic choices. Because ustekinumab is now approved across both pediatric CD and UC, pharmacists may increasingly manage patients transitioning between formulations or coordinating induction infusions with subsequent at-home subcutaneous maintenance dosing. Counseling should emphasize the 2-phase regimen, weight-based intravenous dosing calculations, and the importance of adherence to the 8-week subcutaneous maintenance schedule. Pharmacists should also confirm age-appropriate immunizations are up to date before initiation, since live vaccines should be avoided during treatment.^2,3

Contraindications, Warnings, and Precautions

Ustekinumab is contraindicated in patients with clinically significant hypersensitivity to the drug or its excipients. Boxed warnings and precautions relevant across indications include increased risk of serious infections (including tuberculosis, mycobacterial, and salmonella infections), malignancy risk, serious hypersensitivity reactions including anaphylaxis—some occurring during the first intravenous infusion—posterior reversible encephalopathy syndrome, and noninfectious pneumonia. In the pediatric UC population specifically, the most common adverse reactions were nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhea, sinusitis, fatigue, and nausea.^1,3

Pharmacists should screen for active or latent tuberculosis prior to initiation and counsel patients and caregivers to report signs of infection, unusual bleeding or swelling, or neurologic symptoms promptly.^1,3

With pediatric approvals now secured in both CD and UC, ustekinumab offers clinicians a consistent, non-TNF biologic platform across the pediatric IBD spectrum. Continued postmarketing surveillance and longer-term pediatric UC follow-up data will help clarify durability of response and the drug's comparative place in therapy as treatment algorithms for pediatric IBD continue to evolve.

REFERENCES

1. FDA approves Stelara for pediatric ulcerative colitis. News release. FDA. August 28, 2026. Accessed August 31, 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-stelara-pediatric-ulcerative-colitis

2. Johnson & Johnson submits application to U.S. FDA for Stelara (ustekinumab) in the treatment of pediatric ulcerative colitis. News release. Johnson & Johnson. October 31, 2025. Accessed August 31, 2026. https://www.jnj.com/media-center/press-releases/johnson-johnson-submits-application-to-u-s-fda-for-stelara-ustekinumab-in-the-treatment-of-pediatric-ulcerative-colitis

3. Stelara (ustekinumab) injection, for subcutaneous or intravenous use [prescribing information]. Janssen Biotech, Inc; 2026. Accessed August 31, 2026. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/125261s174lbl.pdf