--Regeneron seeks wider FDA approval for drug Arcalyst in gout patients
--Arcalyst is now on the market for rare disease
--Arcalyst will be reviewed by FDA advisory panel on Tuesday
--Agency is concerned about small treatment effect, possible increased cancer risk
(Adds information on cancer imbalance, and on similar drug from Novartis, in the third, eighth and ninth paragraphs.)
By Jennifer Corbett Dooren
The U.S. Food and Drug Administration said that the treatment effect from a proposed Regeneron Pharmaceuticals Inc. (>> Regeneron Pharmaceuticals Inc) treatment for gout was "small," and the agency raised concerns that the product might increase the risk for cancer.
Regeneron is seeking FDA approval for short-term use of a drug called Arcalyst to treat gout, a form of arthritis. Arcalyst, also known by its generic name rilonacept, is already on the market to treat a rare disease called Cryopyrin-Associated Periodic Syndromes.
The product is an injection that is designed to block a protein called interleukin-1, which is secreted by infection-fighting cells in the body. Too much interleukin-1 can be harmful and is linked with several inflammatory diseases, including gout. But many drugs that block proteins in the body can also increase the risk of serious infections and possibly cancer. The FDA has so far refused to approve a similar drug, Ilaris, from Novartis AG (NVS, NOVN.VX) that Novartis has proposed to use as a long-term treatment in certain gout patients, partly because of concerns about infections.
Regeneron is proposing to use Arcalyst as a short-term, 16-week treatment for gout in combination with other drugs. Gout is caused by a build-up of uric acid, which causes pain, redness, tenderness and a feeling of warmth in joints, often in the joint at the base of the big toe. Patients are treated with a drugs such as allopurinol to lower uric acid. However, during the first few months of treatment, gout can often become worse while uric acid is being lowered. Arcalyst is meant to be used as a temporary treatment while uric-acid levels stabilize.
Arcalyst will be reviewed by the FDA's arthritis drugs advisory committee--made up of non-FDA medical experts--on Tuesday. The FDA released a staff review of the product on Friday in preparation for the meeting.
The agency said clinical studies of the drug showed more patients receiving Arcalyst had a reduction in gout flares compared to those who didn't receive the drug. "However, the treatment effect was small," FDA said. The agency explained that patients in the placebo group had an average of only one gout flare during the 16-week period while those receiving an 80-milligram dose of Arcalyst had about 0.3 flare.
The agency also said that, in the "studied gout population, rilonacept appears to be associated with an increased risk of malignancy," although the FDA also noted that people in the study were already at an increased risk of cancer. The agency said the risk "is small but may be real."
The FDA said there were six cancers in the gout studies seen in patients taking Arcalyst compared to none in the placebo group. In at least two of those cases, Regeneron said, the cancer was diagnosed soon after starting treatment with Arcalyst and it was determined that the cancer had been present before treatment had started.
The agency noted that treatment with the drug lasted only for 16 weeks and most cancers take far longer to develop. However, the FDA said long-term safety data for Arcalyst was lacking.
The panel will be asked to vote on a series of questions such as whether the data submitted support the safety and effectiveness of the drug. The outcome of the votes will amount to a recommendation about whether the panel thinks the FDA should approve the product. The FDA isn't required to follow the advice of its panels but usually does.
Regeneron, in a document prepared for the Tuesday meeting, said the drug is safe and effective for treating gout "and would provide a new option for physicians" to manage the disease.
-By Jennifer Corbett Dooren, Dow Jones Newswires; 202-862-9294; [email protected]