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Vertex-770 potentiator

Question
Dear expert team, my daughters genotype is DF508 and R347P, now that the drug for G551D is going forth for approval because of its effects on the gene do you have any further data to support that this drug would also be effective on other class IV types like R347P as I know it has some effect on gene type R117H.

Any information on this would be greatly received.

Many thanks


Charni
Answer
Dear Charni

Thank you for your excellent question. The drug developed for the G551D mutation is called Ivacaftor it was previously called VX770. Full details are now available in a paper recently published in the New England Journal of Medicine. The drug has gone forward for approval but we do not know as yet if any other mutations will be included in addition to the G551D. It is possible that other gating sometimes known as Class III mutations will be included. However it is unlikely it will be approved for Class IV mutations such as R117H and R347P. There are discussions currently with Vertex to undertake clinical trials in Class IV mutations. These are likely to be short studies and I am optimistic that they will be undertaken in a timely manner.

NEJM 2011: 365; 18

Best wishes

Stuart Elborn Belfast
01.12.2011
The answer is edited by: Prof Stuart Elborn