FDA Expands Indication of Tofacitinib to Include Ulcerative Colitis

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

The approval has made the Pfizer therapy the first oral medicine with an approval of this indication for chronic use—other approved therapies are either intravenously or subcutaneously administered.

Ulcerative colitis impacts more than 900,000 patients in the US, many of which experience the moderately to severely active form. Currently, there is no cure. “New treatments are needed for patients with moderately to severely active ulcerative colitis,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, in a statement. “Today’s approval provides an alternative therapy for a debilitating disease with limited treatment options.”

Tofacitinib was evaluated for the expansion of its indication based on data from 3 clinical trials, including a duo of 8-week, placebo-controlled studies which showed the 10-mg dose of the therapy, when administered twice daily, induced remission in 17% to 18% of patients. In the additional trial, of those who achieved a clinical response after 8 weeks with either twice daily 5-mg or 10-mg doses, remission was induced in 34% and 41% of patients, respectively.

Among the patients achieving remission after 8 weeks on the janus kinase inhibitor, 35% and 47% achieved sustained corticosteroid-free remission when treated with 5 mg and 10 mg, respectively.

The safety profile of the therapy was also explored, with the most common adverse events reported being diarrhea, elevated cholesterol levels, headache, herpes zoster, increased blood creatine phosphokinase, nasopharyngitis, rash and upper respiratory tract infection.

The FDA does not recommend the use of tofacitinib in combination with biological therapies for ulcerative colitis or with potent immunosuppressants, including azathioprine and cyclosporine.

In December 2017, the therapy’s 5mg dose was approved for the treatment of adult patients with active psoriatic arthritis (PsA) who have had inadequate response or intolerance to disease-modifying antirheumatic drugs (DMARDs).

That evaluation was made on data from the phase 3 Oral Psoriatic Arthritis Trial (OPAL) program, featuring 2 pivotal studies—OPAL Broaden, OPAL Beyond—and a long-term extension trial, OPAL Balance.