The US FDA has approved tofacitinib (Xeljanz, Pfizer) for the treatment of moderate-severe ulcerative colitis (UC).
- Janus kinase inhibitor (blocks STAT transcription factor activation and inflammatory signaling)
- First oral agent approved for chronic use in UC
- 10 mg tofacitinib PO BID induced remission in 17-18% of patients (vs. 8.2% placebo) after 8 weeks
- Of patients that responded after 8 week trial, 40.6% who continued 10-mg tofacitinib for 52 weeks achieved sustained remission (versus 11.1% placebo)
- Adverse events (common): arthralgia, diarrhea, elevated cholesterol, headache, shingles, rash, and upper respiratory infection
- Adverse events (severe): malignancy (e.g., lymphoma), serious opportunistic infections (FDA black-box warnings)