Anyone in the UK on Kalydeco?

[lovemyboy507]

Hi I am originally from the UK but have been in the US for a couple of years where we are lucky to have great healthcare insurance and be close to a wonderful cf center. We have the option to move back to the UK (London) but I'm concerned that access to new drugs in the UK is somewhat difficult and don't want my son to suffer because of our decisions. Can anyone give any advice about this or just in general on the CF care in the UK ! Thanks so much

 

[Aboveallislove]

Hey mom,
I'm not in UK, but follow the Vertex stuff pretty closely so might be able to help. First a couple of questions: Is your son currently on Kalydeco and how old? Kalydeco isn't approved for R560T yet, so it wouldn't be paid for in other countries. But with private insurance in U.S. it is possible to get off label if proof it works. My quick research seems to show R560T is in same class as g551 so it should work: http://66roses.blogspot.com/2011/03/what-on-earth-do-f508-r560t-mean.html (I don't have a chance now to dig into that more). If there is evidence it works, you should look into doing off label in U.S. but you won't be able to have that option in EU. Also, in the future the drugs seem to get to patients about 1-2 years sooner in U.S., so Orcambi will be available here in a couple months but not expected to be available to patients in EU until sometime 2016. It is likely a similar pattern will be for other drugs that come down the pipeline for the mutations. Let me know if this doesn't make sense/questions.

 

[lovemyboy507]

Thanks for your reply I will read the link you sent as I thiught R560T was in the same category as dd508 so will check that out! I guess I should give a bit more info, my son is actually to young to get any of the drugs currently or about to come to market but I was interested to see how easy access was as even though a product is launched in the UK lack of NHS funding means patients don't actually get to use these amazing drugs. We don't want to make a huge move back to the UK and find that even though there is a drug for him we can't actually use it as the NHS won't fund.

 

[roeann]

I thought in one of your other notes you said your son was 3.. but I may be mistaken.. per kalydeco.com, KALYDECO is prescribed in patients age 2 years and older

 

[lovemyboy507]

He is nearly 3 but I checked and from what I saw R560t and d508 are both class II so kalydeco wouldn't work - I think the classification in the blog in the link is incorrect. But if I'm wrong please let me know - thanks

 

[roeann]

He is nearly 3 but I checked and from what I saw R560t and d508 are both class II so kalydeco wouldn't work - I think the classification in the blog in the link is incorrect. But if I'm wrong please let me know - thanks

I did a search on pubmed and while I couldn't find anything that specifically said what class R560T is in, sadly it looks like you are correct R560T and d508f have similar impacts on the formation of the CFTR protein (I hope I am phrasing this correctly). However, the new combo drug may help.. I will keep my fingers crossed for you.

 

[lovemyboy507]

Thanks Roeann - there is always the possibility that orkambi may work in other traffick/folding genes and if not their are plenty of others in the pipelines, gotta keep positive and in the mean time like all the other wonderful CF parents we do whatever it takes to keep his lungs as healthy as possible (hence the fact we are basing our move around this!)