Xeljanz/Xeljanz XR Approved for Active Ankylosing Spondylitis

The approval was based on data from a phase 3 study which evaluated tofacitinib in adults with active AS who had an inadequate response to at least 2 NSAIDs.

The Food and Drug Administration (FDA) has approved the supplemental New Drug Application for Xeljanz®/Xeljanz® XR (tofacitinib) for the treatment of adults with active ankylosing spondylitis (AS) who have had an inadequate response or intolerance to 1 or more tumor necrosis factor (TNF) blockers.

The approval was based on data from a multicenter, randomized, double-blind, placebo-controlled phase 3 study (ClinicalTrials.gov Identifier: NCT03502616), which evaluated the efficacy and safety of tofacitinib, a Janus kinase (JAK) inhibitor, in 269 adults with active AS who had an inadequate response to at least 2 nonsteroidal anti-inflammatory drugs. Patients were randomly assigned 1:1 to receive tofacitinib 5mg twice daily or placebo for 16 weeks. 

Results showed that patients treated with tofacitinib achieved greater improvements in Assessment in SpondyloArthritis international Society (ASAS)20 response (primary endpoint) at week 16 compared with placebo (56.4% vs 29.4%; P <.0001). Moreover, 40.6% of patients in the tofacitinib treatment arm achieved an ASAS40 response (secondary endpoint) at week 16 compared with 12.5% of those in the placebo arm (P <.0001). In a subgroup analysis, consistent results were observed among patients who had an inadequate response to TNF blockers.

Tofacitnib was also associated with statistically significant improvements in the components of the ASAS response and other measures of disease activity, including Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) total score, Bath Ankylosing Spondylitis Metrology Index (BASMI), and high sensitivity C-reactive protein (all P <.0001). The safety profile of tofacitinib in patients with AS was consistent with that observed in patients with rheumatoid arthritis and psoriatic arthritis. 

“We are proud to offer Xeljanz, a treatment option for ankylosing spondylitis that does not require an injection or an infusion, to treat this debilitating and chronic immuno-inflammatory disease,” said Mike Gladstone, Global President, Inflammation & Immunology, Pfizer. “This regulatory approval affirms the clinical value and versatility of Xeljanz, the first and only Janus kinase (JAK) inhibitor approved for 5 indications in the United States for the treatment of patients with certain immuno-inflammatory conditions.”

The labeling for Xeljanz/Xeljanz XR has also been updated to include additional warnings on the risks of serious cardiovascular-related events, malignancy, thrombosis, and death. The update follows the FDA’s Drug Safety Communication for certain JAK inhibitors issued on September 1, 2021.


  1. US FDA approves Pfizer’s Xeljanz® (tofacitinib) for the treatment of active ankylosing spondylitis. News release. Pfizer Inc. December 14, 2021. Accessed December 15, 2021. https://www.businesswire.com/news/home/20211214006245/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-XELJANZ%C2%AE-tofacitinib-for-the-Treatment-of-Active-Ankylosing-Spondylitis
  2. Xeljanz. Package insert. Pfizer Inc.; 2021. Accessed December 15, 2021. http://labeling.pfizer.com/showlabeling.aspx?id=959.