J&J submits sBLA for ustekinumab to treat children with ulcerative colitis 2 years and up

On October 31, 2025, Johnson & Johnson submitted a supplemental Biologics License Application (sBLA) to the FDA seeking to expand the approval of ustekinumab (Stelara) for the treatment of children aged 2 years and older with moderately-to-severely active ulcerative colitis (UC).¹

The rare form of inflammatory bowel disease affects approximately 1 million people in the United States, and nearly 20% of those cases occur in children. Pediatric patients often experience more severe and aggressive disease compared to adults, underscoring the need for additional therapeutic options.

“Although there have been significant scientific advances in the treatment of inflammatory bowel diseases, many children with ulcerative colitis still face considerable challenges in managing their condition and limited approved treatment options,” said Chris Gasink, MD, vice president, Medical Affairs, Gastroenterology & Autoantibody, Johnson & Johnson Innovative Medicine.

“The FDA filing of [ustekinumab] in the pediatric population, backed by clinical data and its long-established efficacy and safety profile in existing indications, represents a crucial step toward expanding treatment options and addressing unmet needs in this younger population with relatively few treatments available," added Gasink.

Phase 3 UNIFI Jr trial of ustekinumab in pediatric patients

The FDA submission is supported by data from the Phase 3 UNIFI Jr clinical trial (NCT04630028)—a multicenter, interventional study designed to evaluate the efficacy, safety, and pharmacokinetics of ustekinumab in children with moderately to severely active UC through Week 52.^1,2

The study utilized an open-label intravenous induction phase followed by a randomized, double-blind subcutaneous maintenance phase. Participants who demonstrated a clinical response by Week 8 continued on maintenance therapy and were monitored for sustained response and remission over one year.

According to Johnson & Johnson, UNIFI Jr builds upon the established adult UNIFI program, which previously demonstrated that ustekinumab achieved and maintained clinical remission in adults with UC.

Other pediatric ustekinumab submissions and approved indications

In June 2025, Johnson & Johnson also submitted an sBLA to the FDA seeking approval of ustekinumab for children aged 2 years and older with Crohn’s disease.

Ustekinumab is approved to treat patients aged 6 years and older with active psoriatic arthritis.

Ustekinumab is approved to treat patients 6 years and older with moderate-to-severe psoriasis who could benefit from taking systemic therapies or phototherapy.

References:

1. Johnson & Johnson submits application to US FDA for STELARA (ustekinumab) in the treatment of pediatric ulcerative colitis. Johnson & Johnson. Press release. October 31, 2025. Accessed November 3, 2025. 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
2. A Study of Ustekinumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis (UC) (UNIFI Jr). ClinicalTrials.gov. Updated July 18, 2025. Accessed November 3, 2025. https://www.clinicaltrials.gov/study/NCT04630028