Raptor Pharmaceutical Phase 3 trials with slow release Cysteamine have yielded positive results and the firm is looking to extend the trial as well as filing for marketing approval in the USA and European Union.
Those using Cystagon will be well aware of the limitations it brings – it must be used every 6 hours in order to be effective, but also has side effects such as vomiting and bad breath.
Raptor Pharmaceutical has spent several years developing a slow release formulation of Cysteamine known as DR Cysteamine (RP103). This new drug has an enteric coating – the application of such a coating helps Cysteamine avoid destruction in the stomach and instead reach the small intestine, where research has shown it is better absorbed by the body. The hoped for result is a new drug that need only be taken once every 12 hours, whilst still being potentially more effective than Cystagon. Read The 2010 International Conference Report for more background on this research.
In July 2011, Raptor completed their phase 3 trials of the new drug and we’re pleased to announce their results have been very positive. Despite only taking a DR Cysteamine every 12 hours using a dosage only two thirds the size of a standard Cystagon dosage, the average peak Cystine level in patients was only 0.62 ± 0.05 nmol ½ cystine/mg protein, compared with 0.54 ± 0.5 0.05 nmol ½ cystine/mg protein for Cystagon. This is well within acceptable limits, particularly given the smaller dosage size of the DR Cysteamine.
Raptor now intends to extend the trial as well as filing for regulatory approval in the USA and European Union. The date of such approval is naturally unknown, but we hope it will not be too long, and these are positive results in the field of Cystinosis research.
One consideration of DR Cysteamine is that currently it must be swallowed in large capsule form that may not be suitable for younger children. However, in a separate clinical study, a powdered form of DR Cysteamine has demonstrated bio-equivalence with the capsule form, which could yet lead to this powdered form being a long-term treatment.
Finally, we are also very pleased to announce that the chief medical officer for Raptor Pharmaceutical will be attending the Cystinosis Foundation UK conference to present the results of these phase 3 trials in person.
For more information read Raptor’s full publication.