FDA-Approved Indications for Tofacitinib
Tofacitinib, marketed as Xeljanz, is approved for treating moderate to severe rheumatoid arthritis in adults who have had an inadequate response to methotrexate or other disease-modifying antirheumatic drugs (DMARDs). It is used alone or with methotrexate or other nonbiologic DMARDs.[1]
It is also approved for psoriatic arthritis in adults, alone or combined with methotrexate, and for active ankylosing spondylitis in adults.[1]
In ulcerative colitis, it treats adults with moderate to severely active disease who responded inadequately or were intolerant to TNF blockers.[1]
For polyarticular course juvenile idiopathic arthritis, approval covers patients 2 years and older who failed TNF blockers or other non-TNF biologics.[1]
Approvals in Other Regions
The European Medicines Agency approves tofacitinib for rheumatoid arthritis (with methotrexate), psoriatic arthritis (with or without methotrexate), ankylosing spondylitis, and ulcerative colitis (after TNF or vedolizumab failure). Juvenile idiopathic arthritis approval is limited to patients 10 years and older.[2]
Health Canada matches U.S. approvals for these indications, including juvenile use from age 2.[3]
Off-Label and Investigational Uses
Tofacitinib has been studied off-label for conditions like alopecia areata, atopic dermatitis, and graft-versus-host disease, but lacks broad regulatory approval for these.[4]
Ongoing trials explore Crohn's disease and other inflammatory conditions.
Key Warnings and Boxed Labels
All approvals carry boxed warnings for serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis. Use requires risk assessment, especially in patients with infection risks or cardiovascular factors.[1]
| Indication | Age Group | Combination Allowed? |
|------------|-----------|----------------------|
| Rheumatoid Arthritis | Adults | Yes (with MTX or DMARDs) |
| Psoriatic Arthritis | Adults | Yes (with/without MTX) |
| Ankylosing Spondylitis | Adults | Monotherapy |
| Ulcerative Colitis | Adults | Monotherapy |
| Polyarticular JIA | ≥2 years | After TNF failure |
[1]: FDA Label for Xeljanz (tofacitinib). https://www.accessdata.fda.gov/drugsatfdadocs/label/2023/203214s029lbl.pdf
[2]: EMA Summary for Xeljanz. https://www.ema.europa.eu/en/medicines/human/EPAR/xeljanz
[3]: Health Canada Product Monograph. https://pdf.hres.ca/dpdpm/00060662.PM.pdf
[4]: ClinicalTrials.gov search for tofacitinib (various NCT numbers). https://clinicaltrials.gov