GoutPal Interactive

Pegloticase has been shown to be effective in lowering uric acid in clinical trials, and is aimed at those gout patients who cannot tolerate allopurinol. It uses a genetically engineered form of uricase to breakdown uric acid in the body. This enzyme is common in many animals, but not humans.

The failure of Krystexxa to gain FDA approval means that it will be unavailable to help gout patients for some time, though there may still be access through ongoing clinical trials.

Apparently, Savient have changed the manufacturing process for pegloticase. Resubmission will require either a return to the previous method, or rerunning the clinical trials with the output from the changed process. It is expected that Savient will revert to the original process.

Approval is also dependent on Savient providing:

• A Medication Guide for using Krystexxa safely and effectively.
• A Communication Plan for Savient to correctly notify healthcare providers about infusion risks, life threatening allergic reactions, and risks for patients who also have Glucose-6-phosphate dehydrogenase deficiency (G-6-PD), or heart problems.
• An Assessment Plan for ensuring that the other 2 plans ensure that the risks o Krystexxa are properly communicated to doctor and patient.

Pegloticase is a generic name for PEG-uricase – a genetically engineered form of uricase. Uricase is an enzyme present in some animals that breaks down uric acid in the body. Humans do not produce uricase, so techniques have been developed to produce a type of uricase that humans can use.

Various versions of this treatment are being developed. The one that is most advanced is expected to be approved soon under the brand name Krystexxa, in the USA.

This was recommended for approval in June this year, and the FDA are expected to give final approval on August 1st.

So does this mean you can rush out and buy some in August?

Absolutely not.

Even when production gets up to speed, this is not a gout medication for self-administration. You need to go to your doctor, a clinic, or hospital for pegloticase to be given by intravenous infusion.

I suggest that you discuss this now with your doctor. There may be perfectly acceptable uric acid lowering therapies available to you. We have certainly discussed many other options in the gout forum, from the ubiquitous allopurinol to the newly launched febuxostat (Uloric / Adenuric). Surprisingly, even some blood pressure and cholesterol lowering medicines can have uric acid lowering effects.

If the discussion with your doctor reveals that you are a candidate for pegloticase, then he is in the best position to find a source of the medicine, or refer you to a suitable specialist. He may even know of clinical trials in your area, where you gain access to the treatment prior to it being on general release.

FDA review has been postponed from February to give time for additional analysis of cardiovascular risks.

You can find full details of the meeting, including Savient’s briefing submission on the FDA site.

So what does this mean for gout sufferers?

Assuming approval is given, do not be thinking that you have a newer, easier option for controlling your uric acid.

Krystexxa cannot be taken orally. You get dosed up every two weeks with 8mg delivered by intravenous injection.

As well as being inconvenient, there are fairly high documented reaction rates. On a positve side, it is good that severe gout sufferers have an alternative if they cannot tolerate allopurinol.

So expect plenty drug company stimulated hype over the next few weeks. In fact a gout forum regular has spotted early signs of the latest outbreak of gout hype.

It is such a pity that similar money and exposure does not go into fixing the two great fallacies inherent in healthcare regimes throughout the world:

1. Gout sufferers need better diagnosis. Too many patients are dismissed without gout because “normal” uric acid range is set too high.
2. Allopurinol is rarely prescribed properly. Most gout patients do not get the right dose, the right monitoring, or the right support.

The “shocking” statistics reveal that:

• 65% of adults admitted to being “not at all knowledgeable” about gout.
• Over 70% did not know that gout is a form of arthritis.
• 83% said they have not had their uric acid levels checked in the last five years, or weren’t sure if they had.
• 35% of gout sufferers said they have not had their uric acid levels checked in the last five years, or weren’t sure if they had.
• 49% of adults were not aware of the potential crippling effects of gout, such as needing to use a cane or walker, or having to stop working because of limited mobility.

But where did this data come from, and why does it matter?

It matters, because raising gout awareness might push more people into seeking early treatment. By controlling uric acid at the earliest opportunity, people can avoid or reduce the crippling effects of gout.

Such awareness would be good if it encouraged people to get wise advice on managing uric acid and safe alternatives for pain relief until uric acid was correctly controlled. It would be wonderful if the general practice doctor could be relied on for simple proven therapy that will allow gout patients to keep uric acid below 6mg/dL and live a pain free life.

We should praise the Gout & Uric Acid Education Society for pushing confirmed gout sufferers to seek treatment, and unconfirmed gout patients to seek diagnosis.

Unfortunately, experience (and abundant research) tells us that the doctor we rush to has, in many cases, little more knowledge about gout than we do.

Except, of course, they will have been primed by the manufacturers of recently released Uloric (febuxostat) and soon to be released Krystexxa (pegloticase / PEG-uricase / Puricase). They will know all about the new wonder drugs from Takeda and Savient – the very same pharmaceutical companies who sponsor the Gout & Uric Acid Education Society.