In May 2008 the U.S. based company developing Pegloticase made another upbeat statement assessment of what they have learnt in its Phase 3 and open label extension trials. Note that Pegloticase still awaits approval from medical authorities so I am writing here about a new gout drug that is still under development.

May was a good month for gout sufferers. Febuxostat, (EU trade/brand name Adenuric), the first new major drug medication for gout in over 40 years, was approved for marketing in member countries of the European Union.

What makes Pegloticase a very interesting, and more importantly, a hopeful new gout drug, is the way it works. All drugs used in gout try to cure or alleviate the disease by reducing the body's uric acid (UA) levels. They do this by either inhibiting its production or making it easier to remove.

In the world of natural gout remedies, uric acid's removal can be improved by making it more soluble. This is achieved by drinking lots of water and by raising body pH levels in various ways.

Pegloticase's answer to the excess uric acid level problem is to remove it by ingeniously converting uric acid into something else, a substance called Allantoin. Allantoin is more soluble and better excreted than uric acid. The principle comes from the way most animals do it. They do it by converting UA to Allantoin, but this is something humans cannot do naturally. Hence the formulation of Pegloticase.

Pegloticase is significant because its trials have focused on gout patients who have not had success with other gout drugs, or who have medical conditions which prohibit the use of them. Thus it has been tackling harder-to-treat gout cases, not the easier ones. Hard to treat cases are a significant percentage of the gout sufferer universe.

It has been having a decent degree of success.

Elimination of gout attacks In its most recent statement the developing company's most significant remark was that the extension trial group receiving Pegloticase every two weeks, there were only four gout attacks among those who had been administered Pegloticase (8 mg) every fortnight for two months, in subsequent months. And none in this group had a gout attack after five months of Pegloticase treatment, in following months. All were patients who had been through a phase 3 trial.

In other patient groups there were reductions in attacks and improvement in tophi reduction. Tophi are those knobbly shaped, awkward looking, lumps of MSU crystals that often develop in longer term gout sufferers.

31% of patients who had not reduced tophi at the end of these phase 3 trials did show tophi reduction in the extension trial. It would seem that the longer a gout sufferer is able to take Pegloticase the more successful it may be, in tophi cases at least.

Other encouraging statements were that all extension trial patients who had responded earlier to treatment and who were on the every two week course maintained normal uric acid levels, (some were new joiners). 70% of those on an every four week course who had responded earlier to treatment, maintained normal uric acid levels.

This suggests that progression of the disease may be contained in difficult-to-treat cases, and therefore may not cause other medical conditions such as kidney stones heart disease and hypertension.

Of course Pegloticase has caused side effects, in the extension trial in about 1 in 5 patients. The developing company has said that most were mild, moderate, or controllable. But in some cases more severe side effects have been described as including chest and back pains, muscle cramps, sweating and flushes.

Pegloticase is getting closer to an application for approval by the U.S. FDA. Its developers hope to apply for a Biologics Licence Application in September 2008, and begin marketing it in 2009.