The FDA has approved tofacitinib (Xeljanz, Pfizer) to treat patients with moderate to severely active rheumatoid arthritis who have had inadequate responses to one or more disease-modifying anti-rheumatic drugs (DMARDs).

“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, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.

The approval comes after two large studies recently published in the New England Journal of Medicine showed 51.5% to 65.7% of patients receiving tofacitinib met the American College of Rheumatology criteria of 20% improvement endpoint compared with 26.7% to 28.3% of those given placebo.

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Overall, tofacitinib’s safety and effectiveness has been assessed in seven clinical trials. In all of the trials, patients treated with tofacitinib experienced improvement in clinical response and physical functioning compared to patients treated with placebo.

Results from these studies have also prompted FDA concerns about serious medication side effects, including increased risk for serious infections, tuberculosis, cancers and lymphoma.

As part of a Risk Evaluation and Mitigation Strategy (REMS), tofacitinib will carry a Boxed Warning about these risks. In addition, patients will be provided with a Medication Guide advising them about important safety information, and a communication plan to inform health-care providers about any adverse events experienced in association with tofacitinib.

The most common adverse reactions in clinical trials were upper respiratory tract infections, headache, diarrhea, and inflammation of the nasal passage and the upper part of the pharynx.

The FDA is also requiring a postmarketing study to gather data on the long-term effects of tofacitinib on cardiovascular disease, cancer and serious infections.