U.S. FDA Approves Pfizer’s XELJANZ® (tofacitinib) for the Treatment of Active Ankylosing Spondylitis
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for XELJANZ® / XELJANZ® XR (tofacitinib) for the treatment of adults with active ankylosing spondylitis (AS) who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.
“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 five indications in the United States for the treatment of patients with certain immuno-inflammatory conditions.”
The approval of XELJANZ for AS 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 versus placebo in 269 adult patients living with active AS. The study met its primary endpoint showing that at week 16, the percentage of patients achieving an Assessment in SpondyloArthritis international Society (ASAS)20 response was significantly greater with tofacitinib (56.4%, n= 75) versus placebo (29.4%, n=40) (p<0.0001). In addition, the percentage of patients achieving an ASAS40 response was significantly greater with tofacitinib (40.6%, n=54) versus placebo (12.5%, n=17) (p<0.0001), a key secondary endpoint of the study.1 ASAS20/40 are used for defining improvement or response to treatment.2 The safety profile observed in patients with AS treated with XELJANZ was consistent with the safety profile observed in rheumatoid arthritis (RA) and psoriatic arthritis (PsA) patients.
“Ankylosing spondylitis, a type of arthritis that causes inflammation in certain parts of the spine, affects more than 350,000 people in the U.S.3 This disease often occurs in early adulthood and causes pain, swelling and possibly restricted mobility,”4 said Steven Taylor, Executive Vice President, Mission and Strategic Initiatives of the Arthritis Foundation. “With this approval, physicians and patients now have an additional oral treatment option that can help address this chronic and often progressive disease.”
About XELJANZ® (tofacitinib)
XELJANZ is the first and only oral JAK inhibitor approved in the United States in five indications. XELJANZ is indicated in patients who have had an inadequate response or intolerance to one or more TNF blockers: in adults with active AS, adults with moderately to severely active RA, active PsA, moderately to severe active ulcerative colitis (UC) and in children two and older with active polyarticular course juvenile idiopathic arthritis (pcJIA).
XELJANZ has been studied in more than 50 clinical trials worldwide, including more than 20 trials in RA patients, and prescribed to over 300,000 adult patients (the majority of whom were RA patients) worldwide since 2012.5,6,7 As the developer of XELJANZ, Pfizer is committed to advancing the science of JAK inhibition and enhancing understanding of this medicine through robust clinical development programs in the treatment of immuno-inflammatory conditions.
Earlier this month, the FDA updated the prescribing information for XELJANZ and included a new boxed warning for major adverse cardiovascular events and updated boxed warnings regarding mortality, malignancies and thrombosis (with corresponding updates to applicable warnings and precautions). In addition, indications for the treatment of adults with moderately to severely active RA or active PsA, and patients who are two years of age and older with active pcJIA were revised to require inadequate response or intolerance to one or more TNF blockers.
About Ankylosing Spondylitis
Ankylosing Spondylitis is a chronic, inflammatory disease that affects men and women in early adulthood. The onset of symptoms usually occurs before the age of 30 and seldom occur after the age of 45. Symptoms of AS include chronic pain and stiffness in the back and hips for those living with the disease and can negatively impact health-related quality of life. Over time, some patients may experience fusion of the vertebrae in the spinal column.8 According to studies, more than 350,000 people live with AS in the United States.3
1 Deodhar A, Sliwinska-Stanczyk P, Xu H, Baraliakos X, Gensler LS, Fleishaker D, Wang L, Wu J, Menon S, Wang C, Dina O, Fallon L, Kanik KS, van der Heijde D. Tofacitinib for the treatment of ankylosing spondylitis: a phase III, randomised, double-blind, placebo-controlled study. Ann Rheum Dis. 2021 Apr 27;80(8):1004–13. doi: 10.1136/annrheumdis-2020-219601. Epub ahead of print. PMID: 33906853; PMCID: PMC8292568.
2 Landewé R, van Tubergen A. Clinical Tools to Assess and Monitor Spondyloarthritis. Curr Rheumatol Rep. 2015;17(7):47. doi:10.1007/s11926-015-0522-3
3 Strand V, et al. Prevalence of axial spondyloarthritis in United States rheumatology practices: Assessment of SpondyloArthritis International Society criteria versus rheumatology expert clinical diagnosis. Arthritis Care Res (Hoboken ). 2013;65:1299-1306. doi:10.1002/acr.21994
4 Johns Hopkins Arthritis Center. Ankylosing Spondylitis. Accessed December 1, 2021. https://www.hopkinsarthritis.org/arthritis-info/ankylosing-spondylitis
5 Pfizer Data on File. XELJANZ Worldwide Registration Status.
6 ClinicalTrials.gov. Tofacitinib RA Studies. Accessed June 25, 2020.
7 Pfizer. Data on File. Tofa Counts. April 2019
8 University of Maryland Medical Center. A Patient’s Guide to AS. Accessed August 2021. Available at: https://www.umms.org/ummc/health-services/orthopedics/services/spine/patient-guides/ankylosing-spondylitis
December 16, 2021
