FDA Expands Stelara to Pediatric Ulcerative Colitis; Vedolizumab sBLA Under Review

The Food and Drug Administration (FDA) has expanded the indication for Stelara® (ustekinumab), an interleukin-12 and -23 antagonist, approving it for pediatric patients aged 2 years and older with moderately to severely active ulcerative colitis (UC), in addition to Crohn disease. The agency has also accepted for review a supplemental Biologics License Application (sBLA) for intravenous (IV) Entyvio® (vedolizumab), an integrin receptor antagonist, for the treatment of moderately to severely active UC and Crohn disease (CD) in pediatric patients aged 2 years and older.

The FDA approved Stelara for pediatric ulcerative colitis on August 28, 2026. The approval was supported by evidence from UC studies in adults treated with ustekinumab, pharmacokinetic and safety data from pediatric patients with Crohn disease, and a 52-week study that enrolled 112 UC patients aged 3 to 17 years (UNIFI Jr; ClinicalTrials.gov Identifier: NCT04630028). In the UNIFI Jr study, all patients received a single intravenous administration of ustekinumab at induction week 0. At week 8, study participants (n=109) were randomly assigned to receive ustekinumab subcutaneously every 8 weeks or every 12 weeks for 44 weeks. The primary endpoint was clinical remission at week 8 and at week 52 in those with response to induction at week 8. Findings showed that 79 patients achieved clinical response at week 8. Of these clinical responders, 40.5% (95% CI, 30.4-51.5) achieved clinical remission at week 52. Additional efficacy outcomes at week 52 included:

• Symptomatic remission: 65.8%;
• Endoscopic improvement: 40.5%;
• Clinical remission as assessed using Pediatric Ulcerative Colitis Activity Index score: 64.6%;
• Corticosteroid-free clinical remission: 40.5%; and
• Histologic-endoscopic mucosal improvement: 36.7%.

Ustekinumab was well tolerated in this pediatric UC population, and no new safety signals were identified during the trial.

The FDA accepted Takeda's application for IV Entyvio on June 9, 2026. Entyvio is currently approved for the treatment of active UC and CD in adults. The sBLA for pediatric patients is supported by the phase 3 KEPLER study in UC (ClinicalTrials.gov Identifier: NCT04779307) and the WEBB study in CD (ClinicalTrials.gov Identifier: NCT04779320). A Prescription Drug User Fee Act target date for the first quarter of 2027 has been assigned to the application.

The KEPLER study included patients aged 2 to 17 years with moderately to severely active UC (modified Mayo score of 5-9 with endoscopic subscore ≥2) who had an inadequate response to conventional therapy (ie, steroids, immunomodulators, and/or tumor necrosis factor antagonists). During the induction phase, all participants received IV vedolizumab for 14 weeks, followed by a 40-week, double-blind maintenance phase in which participants who achieved a clinical response at the end of the induction phase (N=93) were randomly assigned to receive either low dose (n=47) or high dose (n=46) IV vedolizumab based on weight. The primary endpoint was clinical remission at week 54 in patients who achieved clinical response following the induction phase. Findings showed 47.3% of patients were in clinical remission at week 54, with 34.7% achieving clinical remission at week 14 (secondary endpoint). Additionally, 29% of patients had attained sustained clinical remission at both week 14 and 54 (secondary endpoint). No new safety signals were identified in this pediatric population. The most common adverse events reported were upper respiratory infection (30%), worsening UC (17.5%), and pyrexia (12.5%).

The ongoing WEBB study is evaluating IV vedolizumab in patients aged 2 to 17 years with moderately to severely active CD who were unresponsive or intolerant to current standard of care. The coprimary endpoints include clinical remission and endoscopic response at week 54.

"The efficacy and safety profile of Entyvio is well-established in adults with more than a dozen years of scientific study and clinical data," said the senior vice president and head of the Gastrointestinal & Inflammation Therapeutic Area Unit. "We're confident that, if approved, Entyvio IV would contribute to addressing the unmet needs in the treatment of pediatric UC and Crohn disease." The study investigator, a pediatric gastroenterologist at MNGI Digestive Health in Minnesota, noted: "Study data showed nearly half of patients were in remission after 1 year and safety was generally consistent with vedolizumab's profile in adults, suggesting that vedolizumab could become important in addressing pediatric UC in those 2 years of age and older." Takeda plans to submit the data to regulatory authorities for review in the hopes of expanding the approval of IV Entyvio to include pediatric UC patients.