Saturday, September 24, 2011

FDA approves Soliris (Eculizumab) for atypical HUS

In a significant development the FDA in the USA approved Soliris (Eculizumab) for use in atypical Hemolytic Uremic Syndrome.

To those saying, "Huh?": Read on!

Atypical Hemolytic Uremic Syndrome (aHUS) is a rare disease that afflicts mainly children and a few adults. It causes kidney failure in a lot of cases. I have that disease and it is the cause of my kidney failure.

For years, there was no treatment for aHUS. Plasmapheresis was the only thing that could be tried and that too with varying success.

Eculizumab, manufactured by Alexion Pharmaceuticals as Soliris is an extremely promising drug that has been found to be useful in the treatment of this disease. For those just diagnosed with it, treatment with this drug can prevent them for going into kidney failure to begin with.

For those like me whose kidneys have been affected by this disease, this drug offers hope of a successful transplant. Without this drug, the chances of a successful transplant for me are a pathetic 20%. With this drug, the chances of success zoom into the heady 90s!

This drug has been FDA approved for treatment in another disease called Paroxysmal Nocturnal Hemoglobinurea for a long time. Only yesterday did the FDA approve it for use in aHUS.

This is a significant moment for the aHUS community as this means the drug will be more widely available.

For me to able to use it, if course, it will need to be available in India, which is another story!

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