Off Label Kalydeco Question

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NatalieB

New member
Jenny, glad I could help!! :) Well I am still fighting the insurance company and now they passed my case onto another Medical Director. Tcfuller, thanks for your response, I feel like you have to get passed a pharmacist, not the medical director to get this approved and your experience makes me think this is true. I didn't actually even think that I would start getting worse once I am off the drug because I have been pretty stable and exercise ALOT to keep myself healthy. My last pill is tonight, and I keep a journal of how I am feeling so I will let you guys know if I start getting worse. Please share any other ideas or experiences to get Kalydeco approved for non G551D mutations, this has already been helpful for me!

Aboveallislove, can you send the link to the study? I usually follow clinicaltrials.gov to see upcoming studies, is this the study you are referring to? http://clinicaltrials.gov/ct2/results?term=vx770&recr=Open the Study who have non G551D Gating Mutations?


I spoke to my doc about this but I do not qualify and he said most likely they will never do studies with my mutations (R347P and L1065P) selected because they are so rare, not enough people to sample... they will most likely study the next set of gating mutations that are the bigger population of CF (which they are doing) and once they prove it works, then they will submit for FDA approval for ALL gating mutations. I just can't wait 2 years for this when I have been on the drug for 8 months now!
 
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NatalieB

New member
Tcfuller, forgot to say... thats amazing that your daughter improved so much, thats tremendous and I wish you the best of luck getting approved again! Keep me posted! One question though, why do you have to get it approved again? did they give you a temporary approval to prove it works and then they will reconsider after x months? I wasn't aware that could happen.
 
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Aboveallislove

Super Moderator
Natalie,
I don't think you are looking at the study I'm thinking of. here is the link:
http://clinicaltrials.gov/ct2/show/NCT01685801?term=vx-770&rank=19

It is a phase 2 just posted a week or two ago. The inclusion criteria are:


Clinical evidence of residual CFTR function based on any 1 of the following: 1)Clinically documented residual exocrine pancreatic function, 2)Sweat chloride value ≤80 mmol/L at screening, or 3) Age of diagnosis ≥12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing

You were diagnosed after 12 (I believe you said) and if you have a gating mutation you have a CFTR mutation associated with residual CFTR function. The study lists several examples (not sure if yours is listed), but the criteria is broad enough to be a catch-all. There is another thread on this newly posted study on who to contact.
 
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tcfuller

New member
NatalieB, thanks for the well wishes for my daughter! Yes, we only got approved on a temp basis for 3 months. I believe the phamacist who helped me must have offered this as a solution to the review team. He personally called me the day I got approval and told me that I would be getting a denial letter, but to ignore it. Sure enough, I got a denial letter the day AFTER we got Kalydeco delivered. So, I'm assuming the team originally denied our request and he helped push it through. I am very nervous right now, quite frankly, about the next approval even though my daughter is doing well. Hoping that if approved this time, it will be on a permanent basis...or at least a longer period of time.

Also, I agree with Aboveallislove. I think the trial she posted may be a good option for you, if you don't get approval right away from your insurance company. This is not first-hand knowledge, but I saw a post on Facebook that said Vertex would pay for travel to Denver if you get chosen for the study. If you are a Facebook user and not already a member, I suggest you join the group "Praying for success with VX 770/809." Lots of good conversations there about many topics related to Kalydeco.

Wishing you all the best. Good luck!
 
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GenH

New member
NatalieB said:
R347P is class 4 gating mutation
Click to expand...

I'm under the impression that R347P is a class 4 conductance mutation (according to the CFGeneE app by Vertex). The class 3 mutations are gating. With R347P some CFTR still gets to the surface, but the channel is narrow so less chloride gets through. So this is where Kalydeco can theoretically help, so that explains why your pfts and sweat chloride improved. R347P is not in the residual function trial (http://www.clinicaltrials.gov/ct2/show/NCT01685801?term=ivacaftor&rank=10) or the gating trial.

I'm guessing your doctor already knows this but I just wanted to make sure your info was accurate when dealing with the insurance company.
 
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baseballfrank

New member
I have 2 copies of F508 and I am on the waiting list for the Phase 3 clinical trial for the combo drug. My question is does anybody have any knowledge of Kalydeco working at all on people with DDF508? I would take ANY effect. I am having a lot of problems with breathing, mucos, asthma etc. that are getting worse and worse. They keep delaying the recruitment for the Phase 3 clinical trial and I am getting frustrated waiting. I can see the carrot, but it's just out of my reach!
 
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marcijo

Guest
Here's a great article I found on R347P: http://ecorn-cf.eu/index.php?id=32&no_cache=1&tx_expertadvice_pi1[showitem]=3190&tx_expertadvice_pi1[search]= - ok-I can't get the link to work right-but if you do a google search on R347P Kalydeco it is the first thing that pops up.

I have R117H - also a class 4 mutation -and I am Kalydeco off label. It has worked amazingly well for me- my sweat test went down from 81 to 36 (my clinic told me that my final number was lower than their patients on G551D!), my x-rays were better, my lung functions are increasing (started at FEV1 of 83% - at least check they were 89% I believe-I go in next month to check again). I rarely cough anymore either. Insurance tried to deny me after being on it for 6 months-but my doctor did a peer to peer call and they (thankfully!) reversed their denial based on my results.
 
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GenH

New member
baseballfrank said:
I have 2 copies of F508 and I am on the waiting list for the Phase 3 clinical trial for the combo drug. My question is does anybody have any knowledge of Kalydeco working at all on people with DDF508? I would take ANY effect. I am having a lot of problems with breathing, mucos, asthma etc. that are getting worse and worse. They keep delaying the recruitment for the Phase 3 clinical trial and I am getting frustrated waiting. I can see the carrot, but it's just out of my reach!
Click to expand...

In the trials I am under the impression that a small group of DDF508 responded to Kalydeco monotherapy. The VX809/Kalydeco combination has had much better results. This is a slide from the recent investor presentation, it does not show Kalydeco alone, but I have heard that an approx 10% improvement in CFTR function was seen. The 2nd generation corrector that is in the slide is one of the next generation of medications, currently being tested in labs.

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