May Vertex Update- VX661 adds further benefit to those on Kalydeco with G551D/F508del

[GenH]

I've just summarised the May 1 Vertex investor conference, includes info about Kalydeco, VX809 & VX661:

http://sixtyfiverosesblog.wordpress.com/2014/05/04/may-2014-vertex-update/

Also, it was announced on May 1 that the trial adding VX661 to patients stable on Kalydeco (with G551D/F508del), showed further improvements in FEV1 (+4.6%) and sweat chloride (-7mmol/L). My blog post above has more info about this. In theory this means that people who are likely respond to Kalydeco (gating, residual function) who also have F508del, may see further benefits when a corrector is added.

 

[jamieharris]

Useful thread. Thanks for sharing

 

[triples15]

Thanks so much for these updates Gen! I appreciate them so much!

Question for ya.. what's the word on the street about the Denver n of 1 trial? I heard that there is not going to be a phase 3 because of lack of participants? Yikes, that's sad. When I went to Denver to be screened last September, the research coordinator told me she believed that residual functions would be added to the label within a year. I was sooo hopeful with that comment. It certainly does not look like that will be the case, if it is not even going to a phase 3. Booo..

You might remember I have S945L, which, on paper, looks to have even more improvement than G551d with Kalydeco alone. So this wait to get it is absolutely killing me. I tried everything to get my doc to prescribe it off label with no luck. Even then would have been an uphill battle with the insurance company.

Anyway, my apologies for the rant. Please let me know if you've heard any underground rumblings about residual functions and where it's going from here.

Thanks a bunch!

Autumn 33 w/Cf

 

[ymikhale]

I am interested too, my dd has residual function mutation

 

[CFParent2]

Thanks so much for these updates Gen! I appreciate them so much!

You might remember I have S945L, which, on paper, looks to have even more improvement than G551d with Kalydeco alone. So this wait to get it is absolutely killing me. I tried everything to get my doc to prescribe it off label with no luck. Even then would have been an uphill battle with the insurance company.
Thanks a bunch!
Autumn 33 w/Cf

We are in the same boat with R117H and DF508. Looks like the FDA should approve for use over 18, but the insurance denied the off label use, stating that the FDA has not yet approved the med for my son's genotype. He coughed most of last night. Seems like the process should take less than 9 months after all the testing is complete. What use is an appeal if the people reviewing the appeal have the most to lose?