The FDA yesterday approved tofacitinib (Xeljanz) for treatment of moderately or severely active rheumatoid arthritis (RA) in patients who have had inadequate results from or are intolerant of methotrexate.
The Pfizer drug, to be taken twice daily, is a so-called JAK inhibitor that works by modifying the important inflammation mediators known as Janus kinases.
The approval is based on evaluations of tofacitinib in seven clinical trials involving adult patients with moderately to severely active RA, all of which showed improvements in clinical response and physical function compared to placebo. "The efficacy of tofacitinib in all reported trials to date is significant when compared to a placebo group with ACR20, 50, and 70 responses after treatment intervals of 12 to 24 weeks" wrote authors from the New York Hospital for Joint Diseases late last year in a review of tofacitinib and other new RA medications, "and promise of improvements has been seen in other areas measuring disease activity ... well as radiographic progression."
However, Xeljanz his associated with an increased risk of serious and opportunistic infections including tuberculosis as well as cancers. The drug has been approved with a Risk Evaluation and Mitigation Strategy (REMS) to inform patients and physicians about these risks, as well as requirements for postmarketing studies to evaluate the long-term risks.
FDA approved the drug two weeks ahead of its own scheduled decision deadline.