FDA Panel Rejects Gout Drug on Safety Concerns

June 21, 2011 -- Safety concerns led an FDA advisory to oppose approval of new drug for gout on Tuesday, despite widespread enthusiasm for the medication's effectiveness against the painful disorder.

The recommendation is likely to push the FDA to hold off on approving the drug, known by the brand name Ilaris, until manufacturers can prove it's safe for long-term use.

The FDA is not required to accept the decision of its advisory panel, but it often does.

Ilaris is an anti-inflammatory drug that studies show can relieve painful gout attacks in patients with gouty arthritis. It's delivered by injection whenever patients have gout flare-ups, which sometimes occur every two to three months or so.

Manufacturers wanted to market the drug, known generically as canakinumab, for gouty arthritis patients who don't get gout relief from other available medications. Novartis, which makes the drug, estimated that about 300,000 U.S. patients would qualify.

Gout occurs when uric acid builds up in the blood, crystallizes, and forms deposits in the joints. It can be extremely painful, and surrounding inflammation can damage adjacent tissue.

Questions About Safety

Studies conducted by manufacturer Novartis showed the drug significantly reduced the pain of gout attacks in most patients and also cut the chances that a gout attack would resurface over the next couple of months.

"We continue to believe in the benefits of [Ilaris] for this painful and debilitating disease and will work closely with the FDA to identify the right patient population who will benefit from this therapy," Trevor Mundel, MD, global head of development at Novartis Pharmaceuticals, says in a news release. "We are encouraged by the committee's enthusiasm and robust discussion and remain committed to addressing the needs of people with gouty arthritis."

The panel voted to reject the drug largely because Novartis only studied the drug for 12 weeks in its two largest trials. But the drug stays in the body for at least a month after each dose, and that left a lot of unanswered questions about how safe Ilaris is when patients take it over the course of several years.

"I found the safety issues to be overwhelmingly concerning," says David Felson, MD, a professor of medicine at Boston University and a member of the advisory panel.

Ilaris suppresses the immune system, and Felson and others worried that patients could be at mounting risk for infections as they take more doses. Novartis did not test Ilaris in older patients or in those with renal failure. Studies also show that the drug elevates uric acid and some forms of cholesterol.

Still, experts acknowledged that gout can be a debilitating problem and that new medications would be welcome for patients who either can't take other drugs or don't respond to them.

"There really is an unmet need in this area. We need something for these patients, says Ted R. Mikulis, an associate professor of medicine at the University of Nebraska and a member of the panel.

"There appeared to be universal enthusiasm for this drug being available," says Kathleen O'Neil, MD an associate professor of pediatrics at the University of Oklahoma College of Medicine and the panel's chair. "We have not met the burden of proof showing that this is a safe drug for that patient population."