FDA Approval Sought for 2 Pediatric Indications for Guselkumab

In July 2017, the U.S. Food & Drug administration (FDA) approved guselkumab (Tremfya) for the treatment of adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy.¹ The FDA subsequently approved guselkumab for the treatment of adults with active psoriatic arthritis (PsA) and moderate to severe active ulcerative colitis. An injectable interleukin (IL) 23 antagonist, guselkumab is administered as a subcutaneous injection every four weeks for most of its current adult indications.² It is not currently approved for pediatric conditions. However, in December 2024, Johnson & Johnson submitted two supplemental biologics license applications (sBLAs) to the FDA for guselkumab for the treatment of:

• Children 5 years and older with active juvenile PsA; and
• Children 6 years and older with moderate to severe plaque psoriasis.³

Background: Juvenile PsA presents as chronic joint inflammation, with swelling and psoriasis. It’s a rare childhood condition affecting about 5% of patients with juvenile idiopathic arthritis (JIA). Children are classified by the International League of Associations for Rheumatology (ILAR) as having juvenile PsA if they have arthritis and psoriasis or if they don’t have psoriatic lesions but do have at least two of the following: dactylitis, nail pitting, onycholysis or a family history of psoriasis in a first-degree relative.⁴

Juvenile PsA differs from adult PsA. In juvenile PsA, skin disease often appears 10 years before the patient develops arthritis, which makes its diagnosis challenging.

Supporting Research

The application for guselkumab as a treatment for juvenile PsA is based on pharmacokinetic extrapolation analyses from two phase 3 PsA studies in adults, DISCOVER 1 (NCT03162796) and DISCOVER 2 (NCT03158285).^5,6 The application also includes efficacy and safety data from the PROTOSTAR (NCT03451851) study.⁷

DISCOVER 1 evaluated the efficacy and safety of subcutaneous guselkumab in participants with active PsA, including those previously treated with one to two tumor necrosis factor inhibitors (TNFi’s). The primary end point was achieving an ACR20 response at week 24. A total of 383 participants were enrolled, of whom 381 received at least one dose of guselkumab. In total, 126 patients received placebo, 127 patients received 100 mg of guselkumab at weeks 0 and 4, then once every eight weeks, and 128 patients received 100 mg of guselkumab once every four weeks.⁵

DISCOVER 2 evaluated the efficacy and safety of subcutaneous guselkumab in adult patients with active PsA who were biologic naive. The study’s primary end point was also the ACR20 response at week 24. A total of 741 participants were randomized into groups, and 739 participants received at least one dose of guselkumab. In the study groups, 246 patients received placebo, 248 patients received 100 mg of guselkumab at weeks 0 and 4, then once every eight weeks, and 245 patients received 100 mg of guselkumab once every four weeks.⁶