Advisory Panel Recommends F.D.A. Approval of Gout Drug NYTimes.com

A federal advisory committee on Tuesday recommended approval of a drug developed by Savient Pharmaceuticals to treat severe cases of gout.

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The 14-to-1 vote for approval reflected a general consensus that the effectiveness of the drug in relieving severe pain and disability outweighed the risk of allergic reactions and suggestions that it could cause cardiovascular problems.

The Food and Drug Administration usually follows the advice of its advisory committees. If it does so in this case, the drug, called Krystexxa, would be the second gout treatment approved this year, after a drought of more than 40 years. Uloric, from Takeda Pharmaceutical, was approved in February.

Gout is an exceedingly painful form of arthritis that afflicts the big toe and other joints. It is sometimes called the “disease of kings” because it can be brought on by a rich diet of red meat, seafood and alcohol that for centuries was available mainly to the wealthy.

With such diets now available to many people, it is estimated that two million to six million Americans have gout. And the number is rising as the population becomes older and heavier.

Savient, based in East Brunswick, N.J., said its drug was intended for only about 50,000 Americans who cannot be treated with other drugs.

In clinical trials, the drug worked for about 40 percent of patients, in some cases bringing marked improvement.

“The effects of this drug are so striking that they can be demonstrated in a small study,” said one committee member, Dr. Milton Packer, a cardiologist at the University of Texas Southwestern Medical School.

But the small size of the studies also made it impossible to determine whether an increase in heart problems among those taking Krystexxa was caused by the drug or was just chance, Dr. Packer and other panel members said. If the drug is approved, they said, further studies should be done on its safety and its use carefully restricted to those not helped by other therapies.

A handful of gout patients testified that the drug had dissolved unsightly and disabling lumps on their bodies, called tophi, and allowed them to walk or use their hands again. Savient paid for hotels and transportation for some patients to attend the meeting, which was in Silver Spring, Md.

“I do not recall participating in any trial where patients were so profoundly transformed by a therapy,” Dr. Herbert Baraf, a Maryland rheumatologist who treated some of the patients in the trials, testified to the committee.

Gout is caused by the buildup of a chemical called uric acid in the blood. Krystexxa, also known as pegloticase, is a form of the enzyme uricase, which breaks down uric acid. Savient licensed worldwide rights to the drug from Duke University and Mountain View Pharmaceuticals of Menlo Park, Calif.

Savient has not said what it will charge for Krystexxa but analysts expect it to amount to tens of thousands of dollars a year. The drug is given by intravenous infusion every two or four weeks.

Regular trading in Savient’s stock was halted on Tuesday, after the shares had closed at .27 on Monday. Shares rose as high as .90 in after-hours trading on Tuesday.