The Food and Drug Administration (FDA) has approved Xeljanz® (tofacitinib; Pfizer) for the treatment of active polyarticular course juvenile idiopathic arthritis (pcJIA) in patients aged 2 years and older.
The approval was supported by data from a phase 3 study that evaluated the efficacy and safety of tofacitinib, a Janus kinase inhibitor, in patients aged 2 to 17 years with active rheumatoid factor (RF) negative polyarthritis, RF positive polyarthritis, extended oligoarthritis, and systemic JIA without systemic manifestations who had an inadequate response or intolerance to at least 1 disease-modifying antirheumatic drug which could have included methotrexate or biologic agents; patients with active juvenile psoriatic arthritis and enthesitis-related arthritis who had an inadequate response to nonsteroidal anti-inflammatory drugs were also included.
The 44-week, 2-part study consisted of an 18-week, open-label, run-in phase followed by a 26-week, double-blind, placebo-controlled, withdrawal phase. Patients received tofacitinib 5mg (or body weight-based equivalent) twice daily during the run-in phase. Of the 225 patients, 76.9% (n=173) patients achieved JIA American College of Rheumatology (ACR) 30 response at week 18 and were randomized into the double-blind phase where they received either tofacitinib or placebo.
The primary end point was the occurrence of disease flare, defined as worsening of at least 30% in 3 or more of the 6 JIA core response variables with no more than 1 of the remaining JIA core response variables improving by at least 30%, at week 44 relative to the double-blind phase baseline at week 18.
Results showed that patients treated with tofacitinib experienced significantly fewer disease flares at week 44 compared with placebo (31% [n=27/88] vs 55% [n=47/85]; difference in proportions: -25%; [95% CI, -39, -10]; P =.0007). The safety profile of tofacitinib was consistent with that seen in adult rheumatoid arthritis patients.
“Although there are already several advanced treatments available, tofacitinib will be an appealing new option given it does not require injections or infusions,” said Dr Hermine Brunner, Director of the Division of Rheumatology at Cincinnati Children’s Hospital Medical Center and Scientific Director of the Pediatric Rheumatology Collaborative Study Group. “These can be quite burdensome to both children with pcJIA and their caretakers.”
Xeljanz is currently supplied as 5mg and 10mg tablets. An oral solution formulation (tofacitinib 1mg/mL) is expected to be available in the first quarter of 2021.
For more information visit pfizer.com.
- US FDA approves Pfizer’s Xeljanz® (tofacitinib) for the treatment of active polyarticular course juvenile idiopathic arthritis. https://www.businesswire.com/news/home/20200928005729/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-XELJANZ%C2%AE-tofacitinib-for-the-Treatment-of-Active-Polyarticular-Course-Juvenile-Idiopathic-Arthritis. Accessed September 29, 2020.
- Xeljanz [package insert]. New York, NY: Pfizer; 2020