The FDA granted approval Tuesday for tofacitinib (Xeljanz) to be used in the treatment of rheumatoid arthritis (RA), in a move that has been highly anticipated by clinicians and patients alike.
The drug is approved for use in patients with moderately to severely active RA who have had inadequate responses to one or more disease-modifying anti-rheumatic drugs (DMARDs).
Tofacitinib, made by Pfizer, is an oral inhibitor of the Janus kinase (JAK) pathway, and is the first new orally administered drug for RA in more than a decade.
"Xeljanz provides a new treatment option for adults suffering from the debilitating disease of RA who have had a poor response to methotrexate," Badrul Chowdhury, MD, PhD, of the FDA said in a statement.
Most of the new RA treatments in recent years have been biologics, such as tumor necrosis factor inhibitors, that are administered subcutaneously or by infusion.
Experts have agreed that patients are likely to welcome the convenience of another oral option.
In May, an FDA advisory committee panel voted 8 to 2 in favor of recommending tofacitinib, agreeing unanimously that the drug is effective, although they had some concerns about safety.
In two large studies published recently in the New England Journal of Medicine, 51.5% to 65.7% of patients receiving the active treatment met the American College of Rheumatology criteria of 20% improvement endpoint compared with 26.7% to 28.3% of those given placebo.
"The clear success of JAK inhibition as a treatment for rheumatoid arthritis, if confirmed by robust long-term efficacy assessed with the use of both clinical and radiographic measures, represents an important clinical advance," David A. Fox, MD, of the University of Michigan in Ann Arbor wrote in an editorial accompanying the two studies.
Efficacy and safety have been assessed in a total of seven trials of patients with active RA.
The FDA advisory panel's safety concerns arising from those trials, included serious infections, seen at a rate of 3 per 100 patient-years, and 12 cases of tuberculosis among patients receiving tofacitinib.
Cases of lymphoma and other cancers also were reported, and the drug's labeling will carry a boxed warning about risks.
The FDA is requiring a postmarketing study to gather further data on potentially serious adverse events, and patients will be given a medication guide advising them on risks and communicating with their caregivers should problems arise.