Ustekinumab Becomes First FDA-Approved Biologic for Pediatric Psoriatic Arthritis

Today, the Janssen Pharmaceutical Companies of Johnson & Johnson announced that their biologic therapy ustekinumab (Stelara) was approved by the US Food and Drug Administration (FDA) for the treatment of active psoriatic arthrtitis (PsA) in pediatric patients 6 years of age and older.

This condition is rare among children and adolescents with chronic inflammatory arthritis, affecting 5-8% of these patient populations. The disease presents similarly in children and adults, with patients typically experiencing joint inflammation and skin lesions, though symptoms can greatly vary.

The challenges that come with treating PsA are amplified in pediatric cases. This FDA decision will aid providers in facilitating an evidence-supported treatment plan based on phase 3b data from the PSTELLAR study.

"We know active pediatric psoriatic arthritis is a challenging inflammatory disease given its rarity and that symptoms, such as swollen joints and skin lesions, can vary significantly in presentation and severity," Terence Rooney, MD, PhD, Vice President, Rheumatology and Maternal Fetal Disease Area, Janssen Research & Development, LLC, said in a statement.

The drug's first approval was in 2009 with an indication for adults with moderate to severe plaque psoriasis (PsO). Now, ustekinumab's profile has been expanded to 4 indications, 2 of which include pediatric patients.

The human monoclonal antibody is the first and only FDA-approved treatment for this particular population. Pharmacokinetic (PK) data from the series of phase 3 studies (PSTELLAR, CADMUS, and CADMUS Jr) supported the decision. The research on adult patients with PsA from PSUMMIT I and II complemented the previously mentioned safety and efficacy data by addressing the rheumatic disease.

"With this pediatric approval of STELARA, we're pleased to help address the unmet needs of these young patients and provide physicians with a much-needed treatment option that has an established track record of safety and efficacy," Davidson continued.

Because of limitations with pediatric patient clinical trials, investigators used an extrapolation approach by reviewing previous pharmacokinetic findings, observing efficacy and safety reports from research investigating pediatric patients with moderate to severe plaque psoriasis who also had active PsA, as well as adult patients affected with the same conditions.

The analyses demonstrated that with ustekinumab, these pediatric PsO patients with active PsA was consistent with that of phase 3 clinical trials of the drug in pediatric PsO patients without active PsA. Additionally, it was found to be successful with adult patients with moderate to severe plaque PsO or adult patients with active PsA. Data on common efficacy endpoints were akin to those observed in these pediatric PsO patients with active PsA.

"The approval of STELARA for use in children six years of age and older with active psoriatic arthritis, which follows the 2020 approval for moderate to severe plaque psoriasis in this population, is complemented by more than 12 years of clinical trial and real-world evidence across all approved indications demonstrating the safety and efficacy of this biologic therapy," said Jennifer Davidson, DO, Vice President of Immunology Medical Affairs, Janssen Scientific Affairs, LLC, stated.