European Commission approves ustekinumab for paediatric Crohn’s disease

Approval extends ustekinumab’s use to younger patients

The European Commission (EC) has approved ustekinumab for the treatment of moderately to severely active Crohn’s disease in paediatric patients weighing at least 40 kg. The decision follows evidence of efficacy and safety in patients who did not respond to conventional or biologic therapies.

Crohn’s disease, while commonly diagnosed in adults, affects around 25% of patients during childhood or adolescence. The onset in younger individuals is linked to severe disease progression, malnutrition, and interference with critical growth periods, which can impact mental health and overall quality of life.

The EC’s approval is based on data from the phase 3 UNITI-Jr clinical study and the REALITI real-world evidence study. The UNITI-Jr trial assessed the safety and efficacy of ustekinumab in paediatric patients over 52 weeks. Results showed 52.1% of patients achieved clinical remission after 8 weeks of treatment, with clinical response observed by week 3.

At 44 weeks, clinical remission rates reached 43.5% and 60.0% among patients treated every 8 weeks or 12 weeks, respectively.

Real-world data from the REALITI study further supported ustekinumab’s safety and effectiveness in routine clinical practice, showing its benefits in both paediatric and young adult populations.

The safety of ustekinumab was examined over extended periods, with the paediatric safety profile consistent with previous adult studies. This approval expands the scope of ustekinumab’s indications, reinforcing its role in managing severe cases of Crohn’s disease and improving paediatric patients’ treatment outcomes.