Pfizer's New Oral Drug for Rheumatoid Arthritis Recommended for Approval

The committee voted 8-2 to recommend approval of the investigational agent tofacitinib, and an FDA decision could come as early as August.

An advisory panel to the FDA voted yesterday on whether or not to recommend approval of the drug tofacitinib, Pfizer’s new oral drug for the treatment of rheumatoid arthritis (RA) in patients who have had inadequate response to 1 or more disease-modifying antirheumatic drugs (DMARDs).

The panel voted that the drug should be approved, but stated that limits should be put into place on the types of patients who should be offered the therapy. Some panel members suggested that patients should only have access to the lower of the 2 proposed dosage strengths for tofacitinib until more safety data are available for the drug.

The FDA has provided an anticipated Prescription Drug User Fee Act (PDUFA) action date of August 2012. If approved by the FDA, tofacitinib would be the first new oral DMARD for RA in more than 10 years and the first RA treatment in a new class of medicines known as Janus kinase (JAK) inhibitors.

Pfizer’s drug works in a different way than other treatments by inhibiting the production of inflammatory mediators. It would be the first oral treatment available for RA if approved.

Although the drug appears to ease the symptoms of RA, the studies did not prove that the treatment halts the progression of the disease, as noted by an article in The New York Times. In addition, there were multiple safety concerns surrounding the use of the drug, including 4 deaths during 1 of the trials. Only 1 of these deaths, according to researchers conducting the trial, was related to tofacitinib.

The risk of serious adverse affects with the drug is not surprising, as many of the drugs used to treat autoimmune diseases carry strong warnings about the increased risk of infections such as tuberculosis, and most of the other drugs used to treat RA are associated with the same risks. John H. Klippel, MD, president of the Arthritis Foundation, told The New York Times, “People who have that disease, in general, are willing to accept the risk of even serious adverse effects from the drugs if they can find drugs that are going to alter the course of the disease.”