Pfizer’s Xeljanz Scores Another FDA-Approved Indication

Drug now approved for moderately to severely active ulcerative colitis

The FDA has approved an additional indication for tofacitinib (Xeljanz, Pfizer). The drug is approved for administration at 10 mg twice daily for at least eight weeks, followed by 5 mg twice daily or 10 mg twice daily, for the treatment of adult patients with moderately to severely active ulcerative colitis (UC).

This approval was based on data from three pivotal phase 3 studies from the Oral Clinical Trials for Tofacitinib in Ulcerative Colitis global clinical development program (OCTAVE Induction 1, OCTAVE Induction 2, and OCTAVE Sustain), and OCTAVE Open, an ongoing open-label, long-term extension study. Data from all three phase 3 studies met their respective primary endpoints, showing a statistically significant, greater proportion of patients in remission at week 8 in the induction studies and in remission at week 52 in the maintenance study in patients with moderately to severely active UC treated with tofacitinib compared with placebo. Full results from OCTAVE Induction 1, OCTAVE Induction 2, and OCTAVE Sustain were published in the New England Journal of Medicine in May 2017.

Risks observed in the UC clinical program include serious infection, including herpes zoster and opportunistic infections, malignancies (including nonmelanoma skin cancer [NMSC] and lymphoproliferative disorders), gastrointestinal perforation, and laboratory abnormalities. There was no discernable difference in frequency of gastrointestinal perforation between the placebo and tofacitinib arms in clinical trials of patients with UC, and many of the trial participants were receiving background corticosteroids.

Dose-dependent adverse reactions seen in patients treated with 10 mg twice daily, compared with 5 mg twice daily, include the following: herpes zoster infections, serious infections, and NMSC.

UC is a chronic and often debilitating inflammatory bowel disease that affects approximately 907,000 people in the U.S. Symptoms of UC can include chronic diarrhea with blood and mucus, abdominal pain and cramping, and weight loss. UC can have a significant effect on work, family, and social activities.

Tofacitinib is the first and only Janus kinase inhibitor approved by the FDA for adult patients with moderately to severely active rheumatoid arthritis, active psoriatic arthritis, and moderately to severely active UC.

Source: Pfizer; May 30, 2018.