An advisory committee to the US Food and Drug Administration on Wednesday urged US regulators to approve a new treatment for rheumatoid arthritis made by the pharmaceutical giant Pfizer.
Tofacitinib, taken orally, could serve as an alternative approach to treating moderate to severe patients who did not respond to one or more traditional therapies such as methotrexate, said the advisory committee.
By a vote of 8-2, the panel urged the drug’s approval despite mixed results on its effects at different doses, and some safety concerns about the risks of cancer — mainly lymphoma — and infections.
Rheumatoid arthritis is a progressive autoimmune disease that causes debilitating joint pain that can lead to deformity, and affects about one percent of people in the United States and Europe, most of them women.
Exactly what causes the disease remains a mystery, and there is no known cure.
According to Herb Baraf, clinical professor of medicine at George Washington University, the safety profile of tofacitinib was very similar to that of a relatively new class of drugs known as biologics.
“It decreases joint pain and swelling. It improves a patient’s sense of well being. It decreases or eradicates morning stiffness,” he told AFP.
“And at the 10 milligram dose it showed that it decreases the destructive nature of the arthritis. It slows down the progression of anatomic changes caused by rheumatoid arthritis.”
Two members of the panel voted “no” and one abstained on the question of whether the safety profile alone was adequate to support approval for the treatment of moderately to severely active rheumatoid arthritis (RA) in patients who have had inadequate response to one or more disease modifying anti-rheumatic drugs (DMARDs), such as methotrexate or leflunomide.
Those panelists said more data was needed to show whether there are safety issues over the long term.
Pfizer spokeswoman Kristen Neese said the company “appreciated the robust discussion.”
She noted that data was presented on “approximately 4,800 tofacitinib-treated patients and one of the largest safety databases ever submitted to the FDA for a new drug application in RA.”
“We believe the clinical trial results demonstrated a favorable benefit/risk profile and support the approval of tofacitinib,” she added.
The FDA does not have to follow the recommendations of the advisory panel though it often does.
People who spoke during the public comment session at the FDA panel’s meeting exprstiffessed support for the drug because it would offer patients, many of whom are frustrated when they do not respond to treatment, another choice.
“This medication will improve the care of our patients who have severe rheumatoid arthritis,” said Daniel Mandel, a doctor with the Coalition for State Rheumatology Organizations.
For Sharon Harris, a nurse who has experienced the pain of rheumatoid arthritis as part of her diagnosis of lupus, the disease caused “a paralyzing pain that literally took my breath away.”
“What works for me may not work for my neighbor, and vice versa,” she said.
[An advisory committee to the US Food and Drug Administration on Wednesday urged US regulators to approve a new treatment for rheumatoid arthritis made by the pharmaceutical giant Pfizer. AFP Photo/Timothy A. Clary]