Pfizer announced that the United States (US) Food and Drug Administration (FDA) approved XELJANZ (tofacitinib) for the treatment of children and adolescents two years and older with active polyarticular course juvenile idiopathic arthritis (pcJIA). Two formulations were approved, a tablet and an oral solution, and are dosed based upon weight. This approval makes XELJANZ the first and only Janus kinase (JAK) inhibitor approved in the US for the treatment of pcJIA.
This approval was based on data from a Phase 3 study including two phases: an 18-week open-label, run-in phase (including 225 patients), followed by a 26-week double-blind, placebo-controlled, randomized, withdrawal phase (including 173 patients) for a total duration of 44 weeks. The study evaluated the efficacy and safety of tofacitinib taken as either a 5 mg tablet or as a 1 mg/mL oral solution twice daily based on the subject’s body weight (<40 kg for the oral solution) and/or patient preference. The trial met its primary endpoint showing that in patients with pcJIA who achieved juvenile idiopathic arthritis (JIA) American College of Rheumatology (ACR) 30 response at the end of the run-in phase, the occurrence of disease flare in patients treated with tofacitinib (31 per cent; n/N=27/88) was statistically significantly (p=0.0007) lower than patients treated with placebo (55 per cent; n/N=47/85) at week 44. In this study, disease flare was defined as a 30 per cent or more worsening in at least three of the six variables of the JIA ACR core set, with no more than one of the remaining JIA core response variables improving by 30 per cent or more (outcome measures used in JIA clinical trials) after randomization,
In general, the types of adverse drug reactions in patients with pcJIA were consistent with those seen in adult rheumatoid arthritis (RA) patients.
XELJANZ oral solution is anticipated to be available by the end of Q1 2021. XELJANZ 5 mg tablets are available immediately.