But the pharmaceutical company behind the therapy was silent regarding potential plans for TGA approval.
The US Food and Drug Administration has approved ustekinumab injections for the treatment of moderately to severely active ulcerative colitis in paediatric patients aged two years and older.
Ustekinumab (Stelara, Janssen Biotech) is the first therapeutic monoclonal antibody to receive FDA approval that does not target tumour necrosis factor alpha (TNFα) for the two primary forms of paediatric inflammatory bowel disease.
The move, announced last month, follows the approval of ustekinumab in the treatment of moderately to severely active Crohn’s Disease in the same population in April of this year. Ustekinumab has received Orphan Drug Designation for the latest indication.
US regulators were convinced by safety and efficacy data for ustekinumab in the treatment of moderately to severely active UC in adult subjects, along with pharmacokinetic and safety data from paediatric patients with CD aged two to 17 years. Additional safety, efficacy, and pharmacokinetic data from a year long study in 112 paediatric patients with UC (aged three to 17 years) also informed the decision-making process.
The paediatric-specific data came from the UNIFI Jr trial, a phase 3 study which showed that a single dose of intravenous ustekinumab led to a clinical response in 79 patients eight weeks later. Of these 79 patients 40.5% achieved clinical remission at week 52, 65.8% were in symptomatic remission, and 40.5% displayed endoscopic improvement.
“[The] remission rate was higher in patients without prior biologic failures (25 [47.2%; 95% CI: 34.4%-60.3%]) compared to those with biologic failure (7 [26.9%; 95% CI: 13.7%-46.1%]),” wrote the researchers.
Common side effects of ustekinumab include nasopharyngitis, headache, abdominal pain, influenza, fever, diarrhoea, sinusitis, fatigue, and nausea.
As of 2 September, there are at least seven products containing ustekinumab that are approved by the TGA for use in Australia. These products are approved for use in adults with moderate to severe plaque psoriasis, active psoriatic arthritis, moderately to severely active CD, and moderately severely active UC. They are also indicated for use in children aged six years and older with moderate to severe plaque psoriasis that is inadequately controlled or unresponsive to other systemic therapies and phototherapy.
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Crohn’s and Colitis Australia CEO Leanne Raven told The Gut Republic that Crohn’s & Colitis Australia and the Gastroenterology Society of Australia had been seeking an expansion of advanced therapy options for patients under the age of 18 since 2024.
“Currently infliximab and adalimumab are available on Pharmaceutical Benefits Scheme for inflammatory bowel disease and ustekinumab specifically for fistulising Crohn’s disease only,” Ms Raven said in a statement. “CCA has advocated to Pharmaceutical Benefits Advisory Committee for access to ustekinumab for children more broadly than its single indication which would be in line with European countries and New Zealand.”
“Children, who are more likely to have severe disease, need treatment options that should not be reliant on industry dependent compassionate access or hospital drug committees. Similarly, Australian children do not have access to vedolizumab and upadacitinib which are funded in other countries such as UK, Canada, and New Zealand.
“Children with active disease have impacts on their physical growth, social development, school attendance, and participation in sport and social activities. Their parents are also affected through work absence, emotional impacts, and financial costs.
“CCA supports widening the eligibility to ustekinumab and other advanced therapies for children with IBD based on clinical discretion.”
Janssen Biotech, the subsidiary of Johnson & Johnson who received the FDA approval for ustekinumab, declined to comment when questioned about their plans to pursue regulatory approval for the monoclonal antibody in Australia.
