Recommendation based on findings from the Phase 3 UNITI-Jr clinical study1 and supported by REALITI real-world evidence study2
Beerse, Belgium (28 February, 2025) – Johnson & Johnson today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended expanding the Marketing Authorisation for STELARA® (ustekinumab) to include the treatment of moderately to severely active Crohn’s disease in paediatric patients weighing at least 40 kg, who have had an inadequate response to, or were intolerant to either conventional or biologic therapy.3
While Crohn’s disease affects the quality of life for all patients,4 it is particularly challenging within the paediatric population,5 as it leads to malnutrition and disrupts critical periods of growth and development, and it is associated with a higher risk of mental health conditions impacting patients’ quality of life.6,7,8
The CHMP recommendation for ustekinumab is based on data from the Phase 3 UNITI-Jr clinical study1. In the UNITI-Jr study, the efficacy and safety of ustekinumab was evaluated in paediatric patients with moderately to severely active Crohn’s disease through 52 weeks of treatment, 8 weeks of induction and 44 weeks of maintenance treatment.1,3 The proportion of paediatric patients weighing at least 40 kg who achieved clinical remission at induction Week 8 was 52.1% (25/48), with clinical response observed as early as Week 3.3 The safety of ustekinumab was studied in one Phase 1 and one Phase 3 trial in paediatric patients with moderately to severely active Crohn’s disease, up to week 240 and week 52, respectively.3 Overall, the safety profile in this cohort (n = 71) was similar to previous studies in adults with Crohn’s disease.3
Results from the real-world evidence REALITI study2 were provided as additional supporting data for the CHMP recommendation. The primary objective of REALITI was to provide evidence for the clinical effectiveness and safety of ustekinumab in routine clinical practice, comparing paediatric patients and young adults.2
The European Commission will review the CHMP recommendation, and a decision is expected later in 2025.
Editor’s Notes
ABOUT THE UNITI-Jr PROGRAMME (EudraCT 2019-004225-24)1
UNITI-Jr is a randomised, double-blind Phase 3 study evaluating the efficacy, safety, and pharmacokinetics of ustekinumab in 48 paediatric patients (aged 2-17) with moderately to severely active Crohn’s disease (defined by a Paediatric Crohn’s Disease Activity Index [PCDAI] score >30) through 52 weeks of treatment (8 weeks of induction and 44 weeks of maintenance treatment).1,3 The study included an open-label induction treatment with a single ustekinumab intravenous dose of approximately 6mg/kg followed by a randomised double-blind subcutaneous maintenance regimen of 90mg administered either every 8 weeks or every 12 weeks.1
ABOUT CROHN’S DISEASE IN PAEDIATRIC PATIENTS
Crohn’s disease is one of the two main forms of inflammatory bowel disease (IBD), which affects over an estimated 1.5 million people across Europe.9 Crohn’s disease within the paediatric population is a rarer form of IBD,5 thought to be triggered through a combination of factors including genetic predisposition, diet or other environmental triggers.10 Symptoms of Crohn’s disease may include abdominal pain, rectal bleeding and weight loss,4 but for the paediatric population, Crohn’s disease can lead to malnutrition and delay growth due to essential nutrients not being properly absorbed by the small intestine.6 In paediatric IBD patients, these chronic symptoms can also lead to higher risk of mental health conditions significantly impacting quality of life.7,8 There is currently no cure for Crohn’s disease.10
ABOUT STELARA® (ustekinumab)
Ustekinumab is a fully human monoclonal antibody and is the first biologic treatment to selectively inhibit the IL-12 and IL-23 pathways.3,11 In the EU, ustekinumab is approved for the treatment of adult patients with moderate to severe CD who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a TNF-α antagonist, or have medical contraindications to such therapies.3 Ustekinumab is also approved for the treatment of adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, or lost response to, or were intolerant to either conventional therapy or a biologic, or have medical contraindications to such therapies.3 In addition to CD and UC, ustekinumab has been approved for the treatment of two further immune-mediated conditions in the EU: plaque psoriasis (Pso) and psoriatic arthritis (PsA).3
ABOUT JOHNSON & JOHNSON
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at www.innovativemedicine.jnj.com or at www.innovativemedicine.jnj.com/emea. Follow us at J&J Innovative Medicine Europe, Middle East & Africa (EMEA). Janssen-Cilag International NV, Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies.
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