FDA approves SNDA for kalydeco

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Robert Fahie

Guest
This is great news. I'm 45 years old and have the R117H mutation and have been taking Kalydeco for the past year. This drug has been a God send. My PFT's have gone up 30%.
 
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Aboveallislove

Super Moderator
Oh, Robert now wonderful. Thanks for sharing. We're still waiting for our sons kalydeco-like drug but I get tears in my eyes every time I read of the miracle it is to someone. Godspeed!
 
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CFParent2

New member
This is awesome news for my son! Although the prescription was denied by UHC twice. They said the R117H mutation was not approved yet. They are making us go through a formal appeal in writing by snail mail. I guess this is SOP for them.

We have been waiting for this type of drug to be discovered for 15+ years. 1-1/2 years for FDA for phase III trial testing, 1 year to get approval. Now we have to wait for the insurance to be gracious enough to update their database and agree to pay for the drug. Their appeal process is supposed to happen within 30 days. I suppose we only have 1 more month to wait, so we are getting close.
 
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Aboveallislove

Super Moderator
Oh mom, I'm so happy for you! We've been waiting for five years for the combo and we are getting close! Please do update how it helps your son....it makes out wait so much more bearable!
 
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CFParent2

New member
Has taken nearly a month for the insurance to overturn their decision not to provide Kalydeco patience with R117h. Had to go through the appeals process. But the good news is that it is supposed to arrive today! Will let you know how things progress since we are not involved in any study.
 
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Aboveallislove

Super Moderator
Smiling ear to ear! I have a favor. If you son has aquagenetic wrinkling could you do before and after pictures? Our sons is sooooo horrible his hands are constantly puffy and even 1 minute in water they start hurting. I'd ove to see what it does! And ou course every little detail! Good lck!
 
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CFParent2

New member
Aboveallislove said:
Smiling ear to ear! I have a favor. If you son has aquagenetic wrinkling could you do before and after pictures? Our sons is sooooo horrible his hands are constantly puffy and even 1 minute in water they start hurting. I'd ove to see what it does! And ou course every little detail! Good lck!
Click to expand...

He did get the medicine. The UPS guy gave them to me as I walked in the door! Took the first dose last night. He said he was breathing better through his nose within an hour, he also had a headace that went away.

This is the first I have heard of aquagenetic wrinkling. I will ask him about it tonight and find out how he slept.
 
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CFParent2

New member
Typically when he starts Tobi, he trys to clear his throat about once every minute, but never produces any sputum. Now he is starting to produce sputum. We are hoping that he is clearing out the bugs that he has cultured for in the past. Also, he played soccer last night and did not have his usual asthma issues. Fortunately he has never had the aquagenetic wrinkling.
 
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