Johnson & Johnson has announced that the European Commission (EC) has approved a 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.
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While Crohn’s disease is more frequently diagnosed in adults, approximately 25% of cases are diagnosed during childhood or adolescence, and is associated with a more extensive and severe disease course over time compared to adults. The onset of Crohn's disease in paediatric patients can be triggered by various factors, including genetic susceptibility, dietary influences or other environmental triggers, leading to malnutrition and interference with critical periods of growth and development, which are associated with increased likelihood of mental health conditions impacting patients’ quality of life.
The EC approval is supported by data from the Phase 3 UNITI-Jr clinical study and the real-world evidence REALITI study. 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. 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. In the maintenance study, at Week 44 43.5% (10/23) and 60.0% (15/25) of paediatric patients receiving ustekinumab 90 mg every 8 weeks (q8w) and every 12 weeks (q12w) achieved clinical remission, with 22.7% (5/22) and 28.0% (7/25) achieving endoscopic response respectively. Results from the real-world evidence REALITI study also supported the EC decision, providing evidence for the clinical effectiveness and safety of ustekinumab in routine clinical practice, comparing paediatric patients and young adults.
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. Overall, the safety profile in this cohort (n = 71) was similar to previous studies in adults with Crohn’s disease.