Regulatory Decision Summary for Stelara

Pediatric psoriasis is a chronic disease associated with serious comorbidities. It has a significant impact on a patients quality of life and social functioning. Many treatment options authorized for adults have not been studied in pediatric groups and guidelines as well as treatment algorithms are based on limited evidence in young children. The pharmacokinetics, safety, and efficacy of Stelara was previously studied (CADMUS) in pediatric patients aged ≥12 to <18 years of age with moderate to severe chronic plaque psoriasis to evaluate the safety and efficacy.

With the CADMUS Jr. Study, the sponsor evaluated the pharmacokinetics, safety, and efficacy of Stelara in the treatment of moderate to severe plaque psoriasis in pediatric patients ≥6 to <12 years of age. This was a phase 3 open label, multicenter study that included patients aged 6 to <12 years of age with a diagnosis of plaque psoriasis for at least 6 months prior to first study drug administration.

The recommended dosage regimen was identical to the regimen used in CADMUS and was the same as is currently authorized for adolescent plaque psoriasis patients. Following the recommended dosage regimen, the pharmacokinetic profile of ustekinumab in children enrolled in this trial, as assessed by serum trough levels, was considered adequately similar to previous data derived from adolescents ≥12 to <18 years of age.

Efficacy evaluation from the study demonstrated a clinically meaningful improvement from baseline in a majority of patients. At week 12, 77.3% of patients achieved a Physicians Global Assessment (PGA) of clear or minimal suggesting overall improvement and reduction in disease burden. This was also supported by the secondary endpoints that demonstrated 75% and 90% improvements in psoriasis area severity index (PASI) scores from baseline, in 84.1% and 63.6% of patients, respectively. Through the study, 77.3% patients reported one or more adverse events (AEs). The most common events were nasopharyngitis, injection-site erythema, pharyngitis, upper respiratory tract infection, tonsillitis, otitis media, and gastroenteritis. There were no deaths or any reports of malignancy in the study. The safety profile of Stelara was comparable to the adolescent population and no new safety signals were identified in the ≥6 to <12-year-old pediatric population.

In the management of pediatric plaque psoriasis in patients ≥6 to <12 years of age, Stelara may provide additional options for healthcare practitioners and caregivers. Overall, Stelara is considered to have a favourable benefit/risk profile for treatment of chronic moderate to severe plaque psoriasis in pediatric patients, who are inadequately controlled by, or are intolerant to, other systemic therapies or phototherapies.