Tofacitinib (Xeljanz) Gets FDA Nod for Ulcerative Colitis

Megan Brooks


May 30, 2018

The US Food and Drug Administration (FDA) has expanded the indication for tofacitinib (Xeljanz, Pfizer) to include adults with moderate to severe active ulcerative colitis.

More than 900,000 adults in the United States have ulcerative colitis, or a chronic inflammation affecting the colon.

Tofacitinib becomes the first oral medicine approved for long-term use in ulcerative colitis. Other FDA-approved treatments for the long-term treatment of moderate to severe active ulcerative colitis must be given by intravenous infusion or subcutaneous injection.

Tofacitinib, a selective oral Janus kinase inhibitor, was approved in 2012 for rheumatoid arthritis  and in 2017 for psoriatic arthritis.  

"New treatments are needed for patients with moderately to severely active ulcerative colitis. Today's approval provides an alternative therapy for a debilitating disease with limited treatment options," Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research, said in an FDA news release.   

The efficacy of tofacitinib for moderately to severely active ulcerative colitis was shown in three controlled clinical trials. Two 8-week, placebo-controlled trials showed that 10 mg of tofacitinib given twice daily induces remission in 17% to 18% of patients by week 8.

In a placebo-controlled trial involving patients who achieved a clinical response by 8 weeks, tofacitinib, given at a dose of 5 mg or 10 mg twice daily, induced remission by 52 weeks in 34% and 41% of patients, respectively. Among patients who achieved remission after 8 weeks of treatment, 35% and 47% achieved sustained corticosteroid-free remission when treated with 5 mg and 10 mg, respectively.

The safety of long-term use of tofacitinib for ulcerative colitis was studied in the 52-week placebo-controlled trial, with additional supportive safety information collected from patients who received treatment in an open-label long-term study.

The most common adverse events associated with tofacitinib for ulcerative colitis are diarrhea, elevated cholesterol levels, headache, herpes zoster, increased blood creatine phosphokinase, nasopharyngitis, rash, and upper respiratory tract infection. Less common serious adverse events include malignancy and serious infections, such as opportunistic infections. Tofacitinib has a boxed warning for serious infections and malignancy.

The FDA said use of tofacitinib in combination with biological therapies for ulcerative colitis or with potent immunosuppressants, such as azathioprine and cyclosporine, is not recommended.

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