Great news

[stephen]

Looks like FDA approved Kalydeco for the additional Residual Function mutations!!!

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm559212.htm

 

[SarahProcter]

For some. Still not approved for every one it works for.

 

[jricci]

I was so happy to hear this wonderful news last week!!
900 more people will hopefully be breathing easier soon.


For those with clinical evidence of residual function who aren’t included in the mutations approved:

Just because you have evidence of residual function, don’t assume that your mutation isn’t included in the triple combo studies. Even though it is for “mutations with minimal CFTR function”, your mutation may be included.
Here are the phase 2 studies that are enrolling:

https://www.cff.org/Trials/Finder/d...mbination-drug-in-people-with-cystic-fibrosis

https://www.cff.org/Trials/Finder/d...mbination-drug-in-people-with-cystic-fibrosis


For those with evidence of residual function who have severe disease and aren’t eligible for clinical trials because your mutation isn’t included and/or your FEV1 is too low or your disease is too unstable to be considered:


There is the remote possibility that you may be able to access Kalydeco/661 through an individual expanded access request. I don’t want to give false hope and I’m not confident at all that Vertex would grant this request, but I needed to mention this in case someone in dire need may have success with it. This is different from the Expanded Access Program for residual function that was mentioned in another thread. It’s not an established “program.”

Your doctor would have to initiate the request and be in complete agreement that you would be a suitable candidate. Here’s some general information about single patient requests for compassionate use/expanded access:

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/#How_To_Apply


I’m really hoping that FDA will soon approve N-of-1 studies so that patients with these rare mutations that have the potential to benefit aren’t left out of clinical trials. But I’m all too aware that some people may not have time to wait.

The other possibility is getting Kalydeco off-label. Here are some threads that may be helpful with this:
http://forums.cysticfibrosis.com/showthread.php/142524-residual-function-amp-Kalydeco-news/page2

http://forums.cysticfibrosis.com/sh...s-Mutations-going-to-be-on-label-for-Kalydeco