Tags: fda | regeneron | arcalyst | gout

FDA Panel Rejects Regeneron's Arcalyst for Gout

Tuesday, 08 May 2012 05:00 PM

Regeneron Pharmaceuticals Inc. failed to win the backing of advisers to the U.S. Food and Drug Administration to expand the use of the autoinflammatory drug Arcalyst to include patients undergoing gout treatments.

An advisory panel voted 11-0 against approval of the injection, citing inadequate safety data, at a meeting Tuesday in Silver Spring, Maryland. The FDA isn’t required to follow the panel’s recommendations. Arcalyst was cleared for sale in February 2008 to treat a rare genetic autoinflammatory disease.

The proposed expanded use of the medication is meant to help patients stay on gout treatments that can cause painful flares of joint pain. FDA staff raised concerns in a May 4 report that Arcalyst’s benefit may be too small to justify a 1 in 244 risk of developing cancer. Arcalyst was linked to six cases of malignancies, including prostate and breast cancers.

“There is a role for this drug or this type of drug,” Lenore Buckley, the chairwoman of the panel and a professor of internal medicine and pediatrics at Virginia Commonwealth University School of Medicine in Richmond, said after the vote. “I just don’t think we have the data yet.”

The FDA has until July 30 to decide on expanded approval for Arcalyst, which would add $128 million to $200 million to Regeneron’s annual sales of the drug by 2015, according to two analyst estimates compiled by Bloomberg. The drug generated $20 million last year, according to data compiled by Bloomberg.

Regeneron rose less than 1 percent to $130.93 at the close in New York. The shares have more than doubled this year.

FDA Discussions

The panel voted 6-5 that Arcalyst works to treat gout flares and 8-3 that Regeneron’s safety data on the treatment is inadequate.

“We are disappointed by the committee’s recommendation,” Tarrytown, New York-based Regeneron said in a statement. “We look forward to further discussions with the FDA.”

The medicine is meant to be taken for 16 weeks and Regeneron only studied the drug for that long. Panel members said cancer isn’t typically associated with a drug such as Arcalyst and want the company to do a longer study to rule out a risk. The panel also wanted to see longer study because some patients likely will use the drug for more than 16 weeks, which Regeneron said is the most-vulnerable period for gout flares.

Regeneron can’t police how long doctors prescribe and patients use the treatment, David Blumenthal, a panel member and assistant professor of medicine at Case Western Reserve University in Cleveland, said at the meeting.

Patient Options

Patients in the placebo group experienced a mean of about one gout flare each over the 16-week treatment period compared with a mean of 0.29 to 0.35 per patient taking Arcalyst. FDA staff also said that Regeneron conducted studies on patients capable of taking other treatments including anti-inflammatories and colchicine.

The panel said Regeneron should possibly limit Arcalyst’s use to patients who can’t take the other therapies.

“There is a small population” that can’t use the other treatment, Tuhina Neogi, associate professor of medicine and epidemiology at the Boston University School of Medicine and a panel member, said during the meeting. “There is an unmet need, but I don’t feel comfortable in the entire gout population.”

Gout treatments include Takeda Pharmaceutical Co.’s Uloric and a generic version allopurinol. They lower levels of uric acid, or bodily waste, that cause gout in excess levels, which manifests through joint pain, according to the National Institutes of Health. Regeneron is seeking approval of Arcalyst to prevent gout flares for people initiating uric-acid lowering therapies.

Allopurinol breaks up uric acid crystals that can cause flares of joint pain that may lead patients to stop taking their medication, the company said. About 750,000 gout patients begin taking allopurinol each year, according to Regeneron.

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Tuesday, 08 May 2012 05:00 PM
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