UPDATE: The FDA in a surprise decision declined to approve, Krystexxa, a gout drug manufactured by Savient Pharmaceuticals of East Brunswick, NJ.

The FDA declined the drug’s approval despite receiving a 14-1 recommendation for approval by its advisory committee in June. Generally, the FDA follows the recommendations of its advisory committees.

Savient plans to resubmit its application for approval Krystexxa early next year.

The FDA’s decision seems to be based on the drugs manufacturing process and not based on its effectiveness, according Savient’s press release.

The agency also concluded that a proposed change in the manufacturing process might make the drug sold to consumers different from the experimental drug tested in the clinical trial.

In addition, the FDA also said that Savient would need to have a program to monitor and reduce the side effects of the drug, particularly allergic reactions and cardiovascular problems.

Krystexxa was developed to treat gout, which is a painful form of arthritis. Gout is increasing because of richer diets, obesity and the aging of society.

The FDA typically does not discuss such regulatory decisions.

For more information please see:

Labels: FDA, Gout, Krystexxa, savient

UPDATE: A federal advisory committee on Tuesday recommended approval of a drug developed by Savient Pharmaceuticals to treat severe cases of gout. The Advisory Committee's recommendation, although not binding, will be considered by the FDA in its review of the Biologics License Application that Savient has submitted for Krystexxa. The FDA usually follows the advice of its advisory committees.

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.

Gout is an exceedingly painful form of arthritis that afflicts the big toe and other joints. It is estimated that two million to six million Americans have gout. Krystexxa is intended for only about 50,000 Americans with gout 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 small size of the studies made it impossible to determine whether an increase in heart problems among those taking Krystexxa was caused by the drug or was just chance. If the drug is approved, the advisory panel recommends further studies on Krystexxa 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.

Though Savient has not named a price for their drug, analysts estimate treatment to cost tens of thousands of dollars a year.

The FDA is expected to make a decision as to whether to grant marketing approval for Krystexxa by August 1, 2009.

For more information please see: http://investor.savient.com/ReleaseDetail.cfm?ReleaseID=390150

Labels: cardiovascular, FDA, Gout, Krystexxa, treatment

The Food and Drug Administration said Friday, June 12, 2009, Krystexxa may successfully treat gout, despite evidence of potentially deadly side effects.

Krystexxa is manufactured by Savient Pharmaceuticals of New Jersey.

Krsytexxa’s new drug application has been under review since December. The FDA already has delayed a decision on the drug once.

Krystexxa is an injectable enzyme designed to lower the body's uric acid levels when administered either once or twice a month. Gout is a condition caused by a buildup of uric acid in the body. The drug appears to reduce gout’s symptoms such as relieving swollen joints and pain flare up. Gout affects about 8 million Americans and is most common in men over 40.

In a clinical study, about 24 percent of patients taking Krystexxa suffered a serious side effect, compared with 12 percent of patients taking a placebo pill. There were six deaths among patients taking the drug compared with three among patients taking placebo, though FDA noted many of them had pre-existing heart conditions.

"Because most of the patients developing the serious cardiovascular adverse events had other cardiovascular risk factors... there was uncertainty concerning whether the cardiovascular adverse events represent a genuine safety signal," states Dr. Bob Rappaport, FDA's director for rheumatology products, in a review posted to the agency's Web site.

The FDA is scheduled to ask a panel of outside arthritis experts next Tuesday to weigh in on the drugs risks and benefits. Though not required not required to follow the group's advice, the FDA generally adopts their recommendations. A final decision on Savient's drug is expected by the end of July.

It is possible that the FDA will consider whether additional studies are needed to evaluate Krystexxa's impact on the heart.

An analyst said the drug can likely win approval so long as the company agrees to limit its use to the target patient population and closely monitor negative side effects. The company is specifically seeking an indication for gout patients who are not receiving relief from other treatments.

For more information: http://www.fda.gov/AdvisoryCommittees/Calendar/ucm155149.htm

Labels: death, drug application, enzyme, FDA, Gout, Krystexxa, savient, uric acid