FDA Approves Tofacitinib for Juvenile Idiopathic Arthritis


Tofacitinib (Xeljanz; Pfizer) is the first and only Janus kinase inhibitor approved in the US for this indication.


The FDA has approved tofacitinib (Xeljanz; Pfizer) for the treatment of children and adolescents 2 years of age and older with active polyarticular course juvenile idiopathic arthritis (pcJIA).

The agency’s approval makes tofacitinib the first and only Janus kinase (JAK) inhibitor approved in the United States for this indication.

Juvenile idiopathic arthritis (JIA) is a chronic inflammatory disease affecting an estimated 300,000 children under the age of 16 in the United States, and characterized as either systemic, oligoarticular, polyarticular, enthesitis-related, psoriatic, or undifferentiated. Patients with pcJIA suffer from arthritis in 5 or more joints, both in small joints of the hands and feet, as well as large joints in the knees, hips, and/or ankles.

Tofacitinib has been previously approved in the US for adults with moderate-to-severe rheumatoid arthritis (RA) after methotrexate failure and adults with moderate-to-severe ulcerative colitis (UC) post tumor necrosis factor inhibitor (TNFi) failure. The drug has been studied in upwards of 50 clinical trials around the world and prescribed to more than 208,000 adult patients within the last 7 years, according to Pfizer.

The approval was based on the results from a phase 3 study comprised of 2 phases: an 18-week open-label, run-in phase that incorporated 225 participants, followed by a 26-week double-blind, placebo-controlled, randomized, withdrawal phase that included 173 participants for a total duration of 44 weeks.

The study collected data on the safety and efficacy of tofacitinib, administered either as a 5 mg tablet or as a 1mg/mL oral solution twice daily based on the patient’s body weight or preference, in which patients under 40 kg received the oral solution. Results showed that patients receiving tofacitinib with pcJIA who achieved a JIA American College of Rheumatology (ACR) 30 response at the conclusion of the run-in phase also demonstrated a statistically significantly lower occurrence of disease flare (31%; n/N=27/88) compared with placebo (55%; n/N=47/85).

“Many children and adolescents living with polyarticular course juvenile idiopathic arthritis, or pcJIA, are in need of advanced oral treatment options, so we are proud to now offer XELJANZ to this patient community,” said Michael Corbo, chief development officer, Inflammation & Immunology, Pfizer Global Product Development. “This approval, which is the fourth indication for XELJANZ, reinforces its utility in the treatment of immune-mediated inflammatory conditions and further demonstrates our expertise in JAK science.”


  1. U.S. FDA Approves Pfizer’s Xeljanz (Tofacitinib) for the Treatment of Active Polyarticular Course Juvenile Idiopathic Arthritis. News Release. Pfizer; September 28, 2020. Accessed September 30, 2020. https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-xeljanzr-tofacitinib-treatment.

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