May 8, 2012  

Proposed Use of Drug Rilonacept for Gout Would Expose Patients to Serious Infections and Possibly Cancer for Minimal Benefits

Public Citizen Tells FDA Arthritis Advisory Committee to Reject Proposed Drug 

WASHINGTON, D.C. – The Food and Drug Administration (FDA) should not approve an immunosuppressant for the prevention of gout flares in adult gout patients because the drug provides only trivial clinical benefits, but exposes patients to a known risk of serious infections and possibly to a slightly increased risk of malignancies and adverse cardiac events, Public Citizen told an FDA advisory committee today. Additionally, other, safer treatments to prevent gout flares are on the market.

The drug, rilonacept, also known as Arcalyst, is manufactured by Regeneron Pharmaceuticals and was approved by the FDA in 2008 only for treating a group of rare serious genetic disorders called cryopyrin-associated periodic syndromes, which cause widespread inflammation in the body. Regeneron now is seeking approval from the FDA to market the drug to prevent acute flares of gout in adult gout patients.

However, the drug offered only meager benefits in clinical trials in gout patients. Although the number of gout flare-ups was reduced in study subjects treated with rilonacept in comparison with subjects taking a placebo, the differences were very small. For example, the drug reduced the average number of gout flares per patient from about one per 16 weeks in patients taking the placebo treatment to 0.3 flare-ups in the same time period in patients getting the drug. Likewise, it reduced the number of days patients experienced moderate to severe pain from gout flares by about 1-2 days over a 16-week period.

In fact, nearly half of subjects in clinical trials treated with the placebo did not have a gout flare, indicating that if rilonacept were approved, 50 percent of patients treated with rilonacept would receive no benefit and only be exposed to the drug’s risks, Public Citizen said.

At the same time, the drug has a known risk of serious infections, and the studies in gout patients raised concern that it may increase the risk of cancer and serious adverse cardiac events in adult gout patients.

“Given that rilonacept offers meager clinical benefits in preventing gout flares in the intended target population, approval of this drug would be justified only if there was certainty that the drug was extremely safe and there was an absence of serious safety signals identified during the clinical development of the drug. But this is not the case,” Dr. Michael Carome, deputy director of Public Citizen’s Health Research Group, told the FDA’s Arthritis Advisory Committee. “In addition, other effective, safer, better studied – and likely significantly cheaper – treatments already are available for preventing and treating the acute flares of gout. Therefore, it would be irresponsible and reckless of the FDA to approve such a potent immunosuppressive drug.”

The same advisory committee in June 2011 rejected a similar drug, canakinumab, for patients who suffer gouty arthritis attacks, because of an unfavorable benefit-risk profile. But in the case of rilonacept, the benefit-risk profile is even worse.

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