FDA Approves Tofacitinib for the Treatment of Active Ankylosing Spondylitis

Officials with the FDA have approved tofacitinib (Xeljanz, Pfizer) 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 blockers.

“Ankylosing spondylitis, a type of arthritis that causes inflammation in certain parts of the spine, affects more than 350,000 people in the US,” said Steven Taylor, executive vice president of mission and strategic initiatives at the Arthritis Foundation, in the press release. “This disease often occurs in early adulthood and causes pain, swelling, and possibly restricted mobility. With this approval, physicians and patients now have an additional oral treatment option that can help address this chronic and often progressive disease.”

Tofacitinib is a Janus kinase (JAK) inhibitor approved for 5 indications in the United States, including for AS, rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and active polyarticular course juvenile idiopathic arthritis. It has been studied in more than 50 clinical trials worldwide, according to the press release.

The approval is based on data from a phase 3, multicenter, randomized, double-blind, placebo-controlled study that evaluated the efficacy and safety of tofacitinib 5 mg twice daily compared with placebo. The trial included 269 adults living with active AS and met its primary endpoints.

At week 16, the percentage of patients achieving an Assessment in SpondyloArthritis International Society (ASAS)20 response was significantly greater with tofacitinib compared with placebo (56.4% and 29.4%, respectively). Furthermore, the percentage of patients achieving an ASAS40 response was also significantly greater with tofacitinib (40.6% versus 12.5%), which was a key secondary endpoint of the study. According to the press release, ASAS20/40 scores are used to define improvement or response to treatment.

The safety profile observed in patients with AS who received tofacitinib was consistent with the safety profile observed in patients with rheumatoid arthritis and psoriatic arthritis, according to the press release.

The FDA has also recently updated the prescribing information for tofacitinib and included a new boxed warning for major adverse cardiovascular events and updated boxed warnings regarding mortality, malignancies, and thrombosis.

“We are proud to offer Xaljanz, a treatment option for ankylosing spondylitis that does not require an injection or an infusion, to treat this debilitating and chronic immune-inflammatory disease,” said Mike Gladstone, global president of inflammation and immunology at Pfizer, in the press release. “This regulatory approval affirms the clinical value and versatility of Xeljanz, the first and only Janus kinase inhibitor approved for 5 indications in the United States for the treatment of patients with certain immune-inflammatory conditions.”

REFERENCE

US FDA Approves Pfizer’s Xeljanz (tofacitinib) for the Treatment of Active Ankylosing Spondylitis. News release. Pfizer; December 14, 2021. Accessed December 15, 2021. https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-xeljanzr-tofacitinib-treatment-0