The U.S. Food and Drug Administration has approved Johnson & Johnson's Simponi (golimumab) for the treatment of children with moderately to severely active ulcerative colitis weighing at least 15 kg, representing the first pediatric indication for this anti-TNF biologic therapy. The approval expands treatment options for a vulnerable patient population where therapeutic choices have been historically limited.
Clinical Trial Results Support Pediatric Approval
The FDA approval is supported by data from the PURSUIT program, which included two multi-center, open-label studies evaluating the efficacy, safety, and pharmacokinetics of subcutaneously administered Simponi in pediatric ulcerative colitis patients. In the pivotal Phase 3 PURSUIT 2 study, 32% of patients achieved the primary endpoint of clinical remission at Week 6.
Secondary endpoints demonstrated additional clinical benefits, with 58% of patients achieving clinical response at Week 6 and 40% showing endoscopic improvement at the same timepoint. Among patients who achieved clinical remission at Week 6, 57% maintained clinical remission of symptoms at Week 54, indicating sustained therapeutic benefit.
The safety profile in pediatric patients was consistent with clinical trials of Simponi in adults with ulcerative colitis, providing reassurance about the therapy's tolerability in younger populations.
Addressing Unmet Medical Need in Pediatric IBD
"Ulcerative colitis is a complex, lifelong condition that is often challenging to manage effectively, particularly for pediatric patients, where relatively few approved options are available," said Chris Gasink, M.D., Vice President, Medical Affairs, Gastroenterology & Autoantibody, Johnson & Johnson Innovative Medicine. "The approval of SIMPONI represents a meaningful milestone, offering a therapy with well-established efficacy and safety along with subcutaneous dosing, for younger patients where treatment options have historically been limited."
The approval addresses a significant clinical need, as more than one million Americans live with ulcerative colitis, with approximately 20% of individuals being pediatric patients. Ulcerative colitis is a form of inflammatory bowel disease characterized by chronic inflammation of the colon, where the immune system inappropriately attacks healthy cells in the colon lining.
Dosing and Administration Guidelines
Simponi is administered as a pre-filled syringe and can be self-administered by patients aged 12 years and older after proper training by a healthcare provider. The dosing regimen is weight-based: pediatric patients weighing at least 40 kg receive 200 mg at Week 0, followed by 100 mg at Weeks 2, 6, and every 4 weeks thereafter. For pediatric patients weighing at least 15 kg to less than 40 kg, the regimen consists of 100 mg at Week 0, followed by 50 mg at Weeks 2, 6, and every 4 weeks thereafter.
Expanding Therapeutic Portfolio
This pediatric approval represents the first for Simponi, which is already approved for four adult indications: moderate to severe rheumatoid arthritis, active psoriatic arthritis, active ankylosing spondylitis, and moderately to severely active ulcerative colitis. The approval demonstrates Johnson & Johnson's commitment to developing treatments for chronic immune-mediated diseases across different age groups.
Simponi works by targeting and blocking TNF-alpha (tumor necrosis factor-alpha), a protein involved in inflammatory processes. The therapy is approved in Europe, Canada, Japan, and other countries for various inflammatory conditions, with Johnson & Johnson maintaining exclusive worldwide marketing rights.
