FDA accepts vedolizumab sBLA for pediatric ulcerative colitis and Crohn's disease

Children and adolescents living with inflammatory bowel disease (IBD) have historically faced a narrow menu of approved therapies, a reality that pediatric gastroenterologists have long flagged as an unmet clinical priority. That landscape may soon change. The FDA has accepted for review Takeda's supplemental Biologics License Application (sBLA) for intravenous (IV) vedolizumab (Entyvio) in pediatric patients ages 2 years and older with moderately to severely active ulcerative colitis (UC) or Crohn's disease.¹ The agency has assigned a Prescription Drug User Fee Act (PDUFA) goal date in the first quarter of calendar year 2027.¹

"A child or teen diagnosed today with UC or Crohn's disease has decades of medical treatment ahead and represents one of the most challenging to treat patient populations in pediatric gastroenterology," said Chinwe Ukomadu, MD, PhD, senior vice president and head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda. "There is a need for additional treatments that can achieve clinical remission."¹ If approved, vedolizumab IV would be the only gut-selective biologic available for this population, according to the company.

KEPLER and WEBB trial design in pediatric UC and Crohn's disease

The sBLA is supported by data from 2 randomized, double-blind, multicenter phase 3 trials enrolling patients ages 2 to 17 years.¹ The KEPLER study (NCT04779307) evaluated vedolizumab IV in pediatric UC; the WEBB study (NCT04779320) is the analogous trial in Crohn's disease and remains ongoing.¹

KEPLER enrolled patients with moderately to severely active UC, defined by a modified Mayo score of 5–9 with an endoscopic subscore of ≥2, who had an inadequate response to conventional therapies including steroids, immunomodulators, and/or tumor necrosis factor (TNF) antagonists.² The study design included a 14-week open-label induction period followed by a 40-week randomized, double-blind maintenance phase comparing 2 dose levels.² The primary endpoint was clinical remission at week 54 among patients who achieved a clinical response after induction.¹ Results announced in February 2026 showed that nearly half (47.3%) of randomized patients met that primary endpoint, with a safety profile generally consistent with the drug's established adult profile.³

For WEBB, co-primary endpoints are clinical remission and endoscopic response at week 54, and full results are anticipated as that trial concludes.¹

Disease burden of pediatric IBD and current treatment gaps

IBD is not a disease of adulthood alone. Approximately 25% of all patients with IBD receive their diagnosis before age 20,¹ and global prevalence among children and adolescents has continued to increase.¹ Pediatric-onset IBD is often more extensive than disease diagnosed in adults, with greater risk for growth impairment, nutritional deficits, and psychosocial consequences.¹

Despite this burden, approved biologic options for children in this age range remain sparse. Current therapies with pediatric IBD indications include TNF antagonists such as infliximab and adalimumab, but a meaningful proportion of patients experience primary non-response or secondary loss of response. No gut-selective biologic has been approved in this age group to date, leaving clinicians with limited mechanistically distinct options when first-line biologics fail.

Vedolizumab mechanism and established adult evidence base

Vedolizumab is an anti-α4β7 integrin monoclonal antibody that selectively blocks the interaction between α4β7 and mucosal addressin cell adhesion molecule-1 (MAdCAM-1), an adhesion molecule predominantly expressed on gut endothelial cells.¹ By preventing lymphocyte trafficking specifically to the gastrointestinal mucosa, vedolizumab exerts a gut-selective anti-inflammatory effect with limited systemic immunosuppression, a pharmacologic profile considered potentially advantageous in a growing pediatric population.

In adults, vedolizumab IV has been approved and in clinical use for more than a decade, with marketing authorization now held in more than 80 countries and subcutaneous formulation authorization in more than 50.¹ Cumulative IV and SC exposure globally exceeds 1.9 million patient-years.¹ This extensive adult safety database provided the pharmacokinetic and safety anchoring used in KEPLER's Bayesian-informed design.

Interpreting the regulatory filing and its limitations

The FDA's acceptance of the sBLA for review is a procedural milestone, not an approval; it confirms the submission was sufficiently complete to warrant a substantive review cycle. The PDUFA target of Q1 2027 sets the agency's review timeline but does not guarantee approval on that date. Clinicians should also note that WEBB data in Crohn's disease are still maturing; the Crohn's arm of the pediatric submission will rest on interim or final WEBB data whose full scope has not yet been publicly reported.

Regulatory review will also need to address dosing extrapolation across the wide age and weight spectrum of patients aged 2 to 17 years, a known complexity in pediatric biologic development. Long-term safety follow-up in this age group beyond the 54-week trial window remains an open question, as does comparative effectiveness against currently approved pediatric biologics.

Takeda has also filed a parallel marketing authorization application with the European Medicines Agency and plans additional submissions in other markets later in 2026.¹

References

1. Takeda Pharmaceutical Company Limited. U.S. FDA accepts Takeda's application for intravenous ENTYVIO® (vedolizumab) in pediatric ulcerative colitis and Crohn's disease. Published June 9, 2026. Accessed June 10, 2026. https://www.takeda.com/newsroom/newsreleases/2026/application-intravenous-entyvio-pediatric/

2. Turner D, Kierkuś J, Korczowski B, et al. Efficacy and safety of intravenous vedolizumab in paediatric patients with moderate-to-severe ulcerative colitis: results from the KEPLER phase 3 trial. J Crohns Colitis. 2026;20(Suppl 1):jjaf231.074. doi:10.1093/ecco-jcc/jjaf231.074

3. Takeda Pharmaceutical Company Limited. Positive phase 3 data demonstrate potential for ENTYVIO® (vedolizumab) to address treatment gap for children and adolescents with moderate to severe ulcerative colitis. Published February 19, 2026. Accessed June 10, 2026. https://www.takeda.com/newsroom/newsreleases/2026/entyvio-phase-3-pediatric-data/

4. Kuenzig ME, Fung SG, Marderfeld L, et al. Twenty-first century trends in the global epidemiology of pediatric-onset inflammatory bowel disease: systematic review. Gastroenterology. 2022;162(4):1147–1159.e4. doi:10.1053/j.gastro.2022.01.004