New gout drug offers hope for those who have exhausted treatment options

Researchers in the U.S. say a new drug effectively treats serious cases of gout - the drug called pegloticase mimics a blood-clearing enzyme missing in humans and helps remove the build up in uric acid, which causes the painful, needle-like crystals to collect around fingers, toes and other joints.

Pegloticase made by the Savient Pharmaceuticals, has successfully completed a phase III trial - the last stage of human testing - which must be done prior to applying for U.S. Food and Drug Administration approval.

The trial showed that uric acid levels fell to target levels among all patients who were given it intravenously, within six hours and in 40% of the cases one infusion kept levels down for at least six months.

Dr. John Sundy, a rheumatologist at Duke University Medical Center in North Carolina, who led the study, says there have been no new drugs for gout for 40 years and there are 50,000 patients in the U.S. alone who will benefit.

Most gout patients are treated with the drug allopurinol but some sufferers don't respond to it or cannot tolerate it and they might benefit from a new therapeutic option.

Gout has often been presented as a disease of men who eat and drink too much, and while it can have links with lifestyle, the disease also has genetic causes.

An estimated 2 million to 3 million people in the United States have the condition.

Dr. Sundy says a typical patient is male, probably in his 40s or 50s, who suddenly develops searing pain in one of his big toes.

The infusions had some side effects however, including back or chest pain, chills, nausea and headaches - Savient Pharmaceuticals is hoping to license the drug under the brand name Puricase.

For the trial the patients, mostly men with an average age of 55 years, were randomly assigned to receive six months of intravenous treatment with either pegloticase or a placebo.

One group of patients received 8 milligrams of pegloticase every two weeks, another group received 8 milligrams of pegloticase every four weeks, and a third group received the placebo.

The researchers say the patients on pegloticase had a significantly better response than those on the placebo and those taking pegloticase also noticed improved physical function.

Dr. Sundy says people with gout suffer from severe pain, increased disability, and reduced quality of life and for those with gout who have exhausted all available treatment options, these findings are exciting.

The researchers presented their findings at the American College of Rheumatology annual meeting, in San Francisco.

Source: http://medschool.duke.edu/