J&J Submits sBLA for Golimumab (Simponi) for Pediatric Ulcerative Colitis

Johnson & Johnson has announced the submission of a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) seeking expanded approval of golimumab (Simponi) for the treatment of children ≥ 2 years of age with moderately to severely active ulcerative colitis (UC).1

The sBLA for golimumab, which is currently approved for the treatment of adults with moderately to severely active UC, is supported by data from the Program of Ulcerative Colitis Research Studies Utilizing an Investigational Treatment (PURSUIT) program, including multi-center, open-label studies evaluating the efficacy, safety, and pharmacokinetics of subcutaneously administered golimumab for the treatment of pediatric UC.1

“This submission marks an important step in addressing the unique and pressing high unmet needs of children and adolescents living with ulcerative colitis, a challenging and lifelong inflammatory disease,” said Esi Lamousé-Smith, MD, PhD, vice president, gastroenterology disease area lead, immunology, Johnson & Johnson Innovative Medicine.1 “At Johnson & Johnson, we are committed to designing clinical programs that prioritize underrepresented populations, including pediatric patients living with immune-mediated diseases, to ensure access to safe and effective treatment options. We look forward to working with the Agency in their review of this SIMPONI® pediatric application.”

Golimumab is a human monoclonal antibody that targets and neutralizes excess TNF-alpha, a protein that can cause inflammation and damage to bones, cartilage, and tissue when overproduced in the body due to chronic inflammatory diseases.1

It was initially approved by the FDA for the treatment of rheumatoid arthritis, psoriatic arthritis, and ankylosing spondylitis in 2009. At the time of its approval for these indications, golimumab was made available in 2 dosage forms, the SIMPONI SmartJect novel autoinjector designed and a prefilled syringe, and was coined as the first patient-administered anti-TNF-alpha therapy to offer an effective once-monthly treatment option.2

Golimumab later earned approval for the treatment of adults with UC who have demonstrated corticosteroid dependence or who have had an inadequate response to or failed to tolerate oral aminosalicylates, oral corticosteroids, azathioprine, or 6-mercaptopurine in 2013.3

The decision was based on data from the PURSUIT clinical trials evaluating patients with moderately to severely active UC who had previously failed or were intolerant to conventional treatments. The approved dose regimen for UC consisted of 200 mg subcutaneously injected at week 0, followed by 100 mg at week 2, and then 100 mg every 4 weeks thereafter.3

Now, Johnson & Johnson is seeking to expand golimumab’s indication in UC to include pediatric patients ≥ 2 years of age. The sBLA is supported by data from the phase 1 PURSUIT Pediatric PK and phase 3 PURSUIT 2 studies, the latter of which is currently in progress to assess the efficacy of golimumab in inducing clinical remission, as assessed by the Mayo score, in pediatric participants with moderately to severely active UC.1

References

1. Johnson & Johnson. Johnson & Johnson submits application seeking U.S. FDA approval of SIMPONI® (golimumab) for the treatment of pediatric ulcerative colitis. December 16, 2024. Accessed December 18, 2024. https://www.jnj.com/media-center/press-releases/johnson-johnson-submits-application-seeking-u-s-fda-approval-of-simponi-golimumab-for-the-treatment-of-pediatric-ulcerative-colitis
2. SIMPONI(TM) (golimumab) Receives FDA Approval as First Once-Monthly Anti-TNF for Treatment of Rheumatoid Arthritis, Psoriatic Arthritis, and Ankylosing Spondylitis. April 24, 2009. Accessed December 18, 2024. https://www.fiercebiotech.com/biotech/simponi-tm-golimumab-receives-fda-approval-as-first-once-monthly-anti-tnf-for-treatment-of
3. Johnson & Johnson. SIMPONI® (GOLIMUMAB) Receives FDA Approval For Ulcerative Colitis. May 16, 2013. Accessed December 18, 2024. https://www.jnj.com/media-center/press-releases/simponi-golimumab-receives-fda-approval-for-ulcerative-colitis