FDA Approves 2 Upadacitinib Formulations for Children with pJIA & PsA

Pediatric rheumatologists now have a new treatment option for children aged 2 years or older who have had an inadequate response to, or demonstrated intolerance to, one or more tumor necrosis factor (TNF) inhibitors. In June, the U.S. Food & Drug Administration (FDA) approved upadacitinib (Rinvoq), a Janus kinase inhibitor, for the treatment of children with active polyarticular juvenile idiopathic arthritis (pJIA) and active psoriatic arthritis (PsA).¹ The FDA also approved a new drug application for the use of 1 mg/mL of upadacitinib (Rinvoq LQ) in an oral solution for the same patient populations.^2,3

The recommended doses of upadacitinib in pediatric patients are weight-based. The doses for the oral solution are:

• 3 mg of upadacitinib twice daily for patients weighing 10 kg to <20 kg;
• 4 mg of upadacitinib twice daily for patients weighing 20 kg to <30 kg; and
• 6 mg of upadacitinib twice daily—or a 15 mg tablet once daily—for patients weighing >30 kg.

Other Indications

In August 2019, the FDA initially approved 15 mg of upadacitinib in extended-release tablets for the treatment of moderate to severe active rheumatoid arthritis (RA) for adults who have had an inadequate response to, or intolerance to, methotrexate.⁴ In December 2021, the FDA expanded the initial indication to include adults with active PsA who have had an inadequate response to, or intolerance to, one or more TNF inhibitors.⁵  

Upadacitinib is also FDA approved for the treatment of:

• Adults and children aged 12 years and older with refractory, moderate to severe atopic dermatitis whose disease is not adequately controlled with other systemic drug products, such as biologics, or when use of those therapies is inadvisable;
• Adults with moderate to severe active ulcerative colitis or Crohn’s disease who have had an inadequate response to, or intolerance to, one or more TNF inhibitors;
• Adults with active ankylosing spondylitis (AS) who have had an inadequate response to, or intolerance to, one or more TNF inhibitors; and
• Adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation who have had an inadequate response to, or intolerance to, TNF inhibitor therapy.⁶

The Research

This latest FDA approval of upadacitinib in pediatric patients is supported by exposure-matched extrapolation of the established efficacy of upadacitinib in patients with RA.⁷ The efficacy and safety of upadacitinib in patients with pJIA was assessed in a multi-center trial in children ages 2 to less than 18 years old (N=83) in an open-label, single-arm study (NCT03725007). At baseline, the study patients had different pJIA subtypes. All patients used weight-based doses of upadacitinib tablets for up to 156 weeks. The study permitted patients who were receiving a stable dose of methotrexate to enter the study, as well as changes to methotrexate dosing during the study. Efficacy was assessed through 48 weeks.