Now that it's through trials an FDA approved, does everyone with the G551D mutation go on it? For anyone who is on it or has a child on it, have you noticd any differences? My kids do not have the G551D mutation, they have DF508 and Q493X. When(if) VX-809 is approved for the DF508 mutation, are we expecting similar results? I have been really keeping an eye out for news on VX-809 and Alturen as well. Thanks guys!
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Now that VX770 is FDA approved...
- Thread starter amber682
- Start date
Now that it's through trials an FDA approved, does everyone with the G551D mutation go on it? For anyone who is on it or has a child on it, have you noticd any differences? My kids do not have the G551D mutation, they have DF508 and Q493X. When(if) VX-809 is approved for the DF508 mutation, are we expecting similar results? I have been really keeping an eye out for news on VX-809 and Alturen as well. Thanks guys!
rubyroselee
New member
I am also interested in hearing how Kalydeco works for the G551Ders. Hopefully people will post on the difference it's made for them once they've been on it. I know that there will likely be some issues with insurance coverage, etc, but I'm sure there will be some who are on it right away. Can't wait to hear about it!
rubyroselee
New member
I am also interested in hearing how Kalydeco works for the G551Ders. Hopefully people will post on the difference it's made for them once they've been on it. I know that there will likely be some issues with insurance coverage, etc, but I'm sure there will be some who are on it right away. Can't wait to hear about it!
J
jessykt
Guest
I'm starting the new drug on Monday...I'll let you know how it goes! I've heard from those on trial and my nurse that people have seen really great results; that may be why it was fast-tracked through FDA approval in just 3 months when it could have taken a year.
J
jessykt
Guest
I'm starting the new drug on Monday...I'll let you know how it goes! I've heard from those on trial and my nurse that people have seen really great results; that may be why it was fast-tracked through FDA approval in just 3 months when it could have taken a year.
MiahsMommy
New member
Ive been keeping my eye out for it too!! Im so excited for the results if these medicines and extremely happy for those who will benefit from it. Soon everyone else will too.. does anyone know if enzymes will still be needed? Nebs cpt's? Will it still be the same daily routine, just extra pills per day?
MiahsMommy
New member
Ive been keeping my eye out for it too!! Im so excited for the results if these medicines and extremely happy for those who will benefit from it. Soon everyone else will too.. does anyone know if enzymes will still be needed? Nebs cpt's? Will it still be the same daily routine, just extra pills per day?
scrapper1264
New member
From what I remember at a meeting a couple of years ago when it was still in Phase 1 or 2, enzymes will still be needed. And it depends on your lung function if nebs/cpts will be needed. That was awhile back though, so things could have changed since then.
scrapper1264
New member
From what I remember at a meeting a couple of years ago when it was still in Phase 1 or 2, enzymes will still be needed. And it depends on your lung function if nebs/cpts will be needed. That was awhile back though, so things could have changed since then.
tomjimdavis
New member
I'm set to start it as soon as it hits my door from the Foundation pharmacy. There have been studies published that within as little as two weeks PFT's have increased as much as 15%. In more than a few cases some test subjects have gained as much as twenty to thirty pounds. @amber682 and @rubyroselee, Vertex is saying if you have the G551D mutation then you'll get it. Foundation Care is selling it at cost and I'm sure most insurance companies would much rather pick up that tab than check out on two or three hospital stays a year. As far as VX-809 is concerned I was unable to find anything that said whether or not the results are expected to be the same. Considering that they are alike in theory, not function, that is to treat the root of the problem, we can only hope that the results will be very much alike. It'll be interesting to see what happens when the two are paired to treat those like @jessykt with the heterozygous DF508 and G551D mutations. @MiahsMommy, because the drug's still in its infant stages, I've gathered, it isn't known what maintenance meds we'll still have to continue doing. More than likely we will stay the same course we're on. Hopefully, TOBI will find it's way out of our typical day, due to fewer exacerbations and infections. It's important to remind ourselves that although it's the closest thing to a cure, it isn't. But it is certainly the best time in the history of this disease to be ailed by it. My family and many others have been robbed of precious lives. I feel blessed but ultimately undeserving that I'm one of the few 4% that will be receiving it. I wish your families well.
tomjimdavis
New member
I'm set to start it as soon as it hits my door from the Foundation pharmacy. There have been studies published that within as little as two weeks PFT's have increased as much as 15%. In more than a few cases some test subjects have gained as much as twenty to thirty pounds. @amber682 and @rubyroselee, Vertex is saying if you have the G551D mutation then you'll get it. Foundation Care is selling it at cost and I'm sure most insurance companies would much rather pick up that tab than check out on two or three hospital stays a year. As far as VX-809 is concerned I was unable to find anything that said whether or not the results are expected to be the same. Considering that they are alike in theory, not function, that is to treat the root of the problem, we can only hope that the results will be very much alike. It'll be interesting to see what happens when the two are paired to treat those like @jessykt with the heterozygous DF508 and G551D mutations. @MiahsMommy, because the drug's still in its infant stages, I've gathered, it isn't known what maintenance meds we'll still have to continue doing. More than likely we will stay the same course we're on. Hopefully, TOBI will find it's way out of our typical day, due to fewer exacerbations and infections. It's important to remind ourselves that although it's the closest thing to a cure, it isn't. But it is certainly the best time in the history of this disease to be ailed by it. My family and many others have been robbed of precious lives. I feel blessed but ultimately undeserving that I'm one of the few 4% that will be receiving it. I wish your families well.
J
jipleary
Guest
This is just what I understand about the drugs from information I've gathered through the internet and stuff. Feel free to correct me or add info etc......... But this is how I understand it in layman's terms.
CF has a malfunction in the "doors" that regulate salt balances in all the cell in the body. (Simply put). The G551D has the doors but they don't open properly. Kalydeco allows the doors to function properly and thus the salt balance in the body evens out and eventually the mucus disappears. Of course this is all in theory and nobody knows for sure what will happen to each patient and they don't even really know the long term effects of the drug. But whatever, it's still progress!!!!
In the the DF508 mutation these "doors" aren't even fully developed to be able to function properly. The idea is that people with the DF508 gene would be able to take another drug (I think this is the VX-809) and this would develop the doors in the cells and then Kalydeco would be able to make them function properly. Or maybe VX-809 includes both steps, I'm not sure. In terms of expected results, in theory it should work great just like Kalydeco works for G551D patients but I have no idea about the actual results. Hopefully it works just as well.
Either way it's exciting and progress and research is wonderful.
So thats what I think I know. But the more you know the more you don't know.
Questions? Corrections?
CF has a malfunction in the "doors" that regulate salt balances in all the cell in the body. (Simply put). The G551D has the doors but they don't open properly. Kalydeco allows the doors to function properly and thus the salt balance in the body evens out and eventually the mucus disappears. Of course this is all in theory and nobody knows for sure what will happen to each patient and they don't even really know the long term effects of the drug. But whatever, it's still progress!!!!
In the the DF508 mutation these "doors" aren't even fully developed to be able to function properly. The idea is that people with the DF508 gene would be able to take another drug (I think this is the VX-809) and this would develop the doors in the cells and then Kalydeco would be able to make them function properly. Or maybe VX-809 includes both steps, I'm not sure. In terms of expected results, in theory it should work great just like Kalydeco works for G551D patients but I have no idea about the actual results. Hopefully it works just as well.
Either way it's exciting and progress and research is wonderful.
So thats what I think I know. But the more you know the more you don't know.
Questions? Corrections?
J
jipleary
Guest
This is just what I understand about the drugs from information I've gathered through the internet and stuff. Feel free to correct me or add info etc......... But this is how I understand it in layman's terms.
CF has a malfunction in the "doors" that regulate salt balances in all the cell in the body. (Simply put). The G551D has the doors but they don't open properly. Kalydeco allows the doors to function properly and thus the salt balance in the body evens out and eventually the mucus disappears. Of course this is all in theory and nobody knows for sure what will happen to each patient and they don't even really know the long term effects of the drug. But whatever, it's still progress!!!!
In the the DF508 mutation these "doors" aren't even fully developed to be able to function properly. The idea is that people with the DF508 gene would be able to take another drug (I think this is the VX-809) and this would develop the doors in the cells and then Kalydeco would be able to make them function properly. Or maybe VX-809 includes both steps, I'm not sure. In terms of expected results, in theory it should work great just like Kalydeco works for G551D patients but I have no idea about the actual results. Hopefully it works just as well.
Either way it's exciting and progress and research is wonderful.
So thats what I think I know. But the more you know the more you don't know.
Questions? Corrections?
CF has a malfunction in the "doors" that regulate salt balances in all the cell in the body. (Simply put). The G551D has the doors but they don't open properly. Kalydeco allows the doors to function properly and thus the salt balance in the body evens out and eventually the mucus disappears. Of course this is all in theory and nobody knows for sure what will happen to each patient and they don't even really know the long term effects of the drug. But whatever, it's still progress!!!!
In the the DF508 mutation these "doors" aren't even fully developed to be able to function properly. The idea is that people with the DF508 gene would be able to take another drug (I think this is the VX-809) and this would develop the doors in the cells and then Kalydeco would be able to make them function properly. Or maybe VX-809 includes both steps, I'm not sure. In terms of expected results, in theory it should work great just like Kalydeco works for G551D patients but I have no idea about the actual results. Hopefully it works just as well.
Either way it's exciting and progress and research is wonderful.
So thats what I think I know. But the more you know the more you don't know.
Questions? Corrections?
S
SarahProcter
Guest
The early results from preliminary studies of VX809 were nowhere near as exciting as the early results of VX770 within its target population. This does not mean that subsequent studies of VX809 for different doses at different durations, and perhaps also in combination with different doses of VX770, may not yield more impressive results; but it does mean that what we've seen so far hasn't been game-changing, the way that VX770 for G551D does appear to be truly game-changing.
VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.
VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.
S
SarahProcter
Guest
The early results from preliminary studies of VX809 were nowhere near as exciting as the early results of VX770 within its target population. This does not mean that subsequent studies of VX809 for different doses at different durations, and perhaps also in combination with different doses of VX770, may not yield more impressive results; but it does mean that what we've seen so far hasn't been game-changing, the way that VX770 for G551D does appear to be truly game-changing.
VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.
VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.
J
jipleary
Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> The early results from preliminary studies of VX809 were nowhere near as exciting as the early results of VX770 within its target population. This does not mean that subsequent studies of VX809 for different doses at different durations, and perhaps also in combination with different doses of VX770, may not yield more impressive results; but it does mean that what we've seen so far hasn't been game-changing, the way that VX770 for G551D does appear to be truly game-changing. VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.</end quote>
This.
This.
J
jipleary
Guest
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>SarahProcter</b></i> The early results from preliminary studies of VX809 were nowhere near as exciting as the early results of VX770 within its target population. This does not mean that subsequent studies of VX809 for different doses at different durations, and perhaps also in combination with different doses of VX770, may not yield more impressive results; but it does mean that what we've seen so far hasn't been game-changing, the way that VX770 for G551D does appear to be truly game-changing. VX661 is another candidate corrector, similar in concept to VX809. Early testing for it should occur this year.</end quote>
This.
This.