SUPPORTING INDIVIDUALS, FAMILIES AND RESEARCHERS
IN THE CYSTINOSIS COMMUNITY

Raptor Phase 3 Trials Begin

Raptor Pharmaceuticals in the USA have completed the recruitment of 30 patients for phase three trials of their new slow release Cysteamine-based drug that they hope will offer a significant improvement to the lives of Cystinotics. The trials will last for eight weeks and Raptor hope to have the results available by the end of quarter one of 2011. It is not known when the new drug will actually hit the market.

The new treatment is based on an enteric coating that means the active component of the medicine is able to reach the small intestine, where it is more effectively absorbed by the body. Early trials had shown significantly improved results in Cystinotic patients taking the drug every 12 hours, compared to every 6 hours for Cystagon (the current main treatment for Cystinosis).

This is an exciting period for the Cystinosis community, but it should be remembered that the drug is still in the trial phase and it could be some time before the treatment is released.

You can read more at Raptor’s website.

For more background on Raptor’s work, see our 2010 International Conference Report.

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