VX-770

jbrandonAW · Aug 7, 2009

The Cystic Fibrosis Foundation depends on people like you to fund lifesaving CF research!

Dear Friend,

We've reached an exciting milestone in the search for a cure.

One of our most promising potential therapies, VX-770, is moving forward in clinical trials.

This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.

VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.

To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today's Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.

This milestone would not be possible without your support, and we need your help - now more than ever - to make this drug a reality.

If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.

You can also help support this and other lifesaving research by making a donation today.
Donate Now

Together we can make CF stand for "cure found."

Thank you for your support,

Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
800-FIGHT-CF
info@cff.org
www.cff.org 

This is an email I recieved today. My doc said to me that it could be out by the end of next year!!! So exciting I could almost cry!

jbrandonAW · Aug 7, 2009

The Cystic Fibrosis Foundation depends on people like you to fund lifesaving CF research!

Dear Friend,

We've reached an exciting milestone in the search for a cure.

One of our most promising potential therapies, VX-770, is moving forward in clinical trials.

This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.

VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.

To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today's Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.

This milestone would not be possible without your support, and we need your help - now more than ever - to make this drug a reality.

If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.

You can also help support this and other lifesaving research by making a donation today.
Donate Now

Together we can make CF stand for "cure found."

Thank you for your support,

Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
800-FIGHT-CF
info@cff.org
www.cff.org 

This is an email I recieved today. My doc said to me that it could be out by the end of next year!!! So exciting I could almost cry!

jbrandonAW · Aug 7, 2009

The Cystic Fibrosis Foundation depends on people like you to fund lifesaving CF research!

Dear Friend,

We've reached an exciting milestone in the search for a cure.

One of our most promising potential therapies, VX-770, is moving forward in clinical trials.

This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.

VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.

To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today's Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.

This milestone would not be possible without your support, and we need your help - now more than ever - to make this drug a reality.

If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.

You can also help support this and other lifesaving research by making a donation today.
Donate Now

Together we can make CF stand for "cure found."

Thank you for your support,

Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
800-FIGHT-CF
info@cff.org
www.cff.org 

This is an email I recieved today. My doc said to me that it could be out by the end of next year!!! So exciting I could almost cry!

jbrandonAW · Aug 7, 2009

The Cystic Fibrosis Foundation depends on people like you to fund lifesaving CF research!

Dear Friend,

We've reached an exciting milestone in the search for a cure.

One of our most promising potential therapies, VX-770, is moving forward in clinical trials.

This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.

VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.

To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today's Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.

This milestone would not be possible without your support, and we need your help - now more than ever - to make this drug a reality.

If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.

You can also help support this and other lifesaving research by making a donation today.
Donate Now

Together we can make CF stand for "cure found."

Thank you for your support,

Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
800-FIGHT-CF
info@cff.org
www.cff.org 

This is an email I recieved today. My doc said to me that it could be out by the end of next year!!! So exciting I could almost cry!

jbrandonAW · Aug 7, 2009

The Cystic Fibrosis Foundation depends on people like you to fund lifesaving CF research!
 
 
 
 
 
 
 
 Dear Friend,
 
 We've reached an exciting milestone in the search for a cure.
 
 One of our most promising potential therapies, VX-770, is moving forward in clinical trials.
 
 This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.
 
 VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.
 
 To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today's Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.
 
 This milestone would not be possible without your support, and we need your help - now more than ever - to make this drug a reality.
 
 If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.
 
 You can also help support this and other lifesaving research by making a donation today.
 Donate Now
 
 Together we can make CF stand for "cure found."
 
 Thank you for your support,
 
 Robert J. Beall, Ph.D.
 President and CEO
 Cystic Fibrosis Foundation
 800-FIGHT-CF
 info@cff.org
 www.cff.org 
 
 This is an email I recieved today. My doc said to me that it could be out by the end of next year!!! So exciting I could almost cry!

RonnieSharpe · Aug 8, 2009

What is the timeline for getting VX-770 compound into patients' hands?

It is too early to speculate on this. It takes several years for a promising drug to make it from trials to the pharmacy shelf. We are going as fast as we can, but additional trials are necessary before we know whether this promising compound will be approved by the Food and Drug Administration (FDA).

**Taken directly from the CFF website:<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/**
">http://www.cff.org/research/Cl...search/FAQs/VX-770/**
</a>
Ronnie

RonnieSharpe · Aug 8, 2009

What is the timeline for getting VX-770 compound into patients' hands?

It is too early to speculate on this. It takes several years for a promising drug to make it from trials to the pharmacy shelf. We are going as fast as we can, but additional trials are necessary before we know whether this promising compound will be approved by the Food and Drug Administration (FDA).

**Taken directly from the CFF website:<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/**
">http://www.cff.org/research/Cl...search/FAQs/VX-770/**
</a>
Ronnie

RonnieSharpe · Aug 8, 2009

What is the timeline for getting VX-770 compound into patients' hands?

It is too early to speculate on this. It takes several years for a promising drug to make it from trials to the pharmacy shelf. We are going as fast as we can, but additional trials are necessary before we know whether this promising compound will be approved by the Food and Drug Administration (FDA).

**Taken directly from the CFF website:<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/**
">http://www.cff.org/research/Cl...search/FAQs/VX-770/**
</a>
Ronnie

RonnieSharpe · Aug 8, 2009

What is the timeline for getting VX-770 compound into patients' hands?

It is too early to speculate on this. It takes several years for a promising drug to make it from trials to the pharmacy shelf. We are going as fast as we can, but additional trials are necessary before we know whether this promising compound will be approved by the Food and Drug Administration (FDA).

**Taken directly from the CFF website:<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/**
">http://www.cff.org/research/Cl...search/FAQs/VX-770/**
</a>
Ronnie

RonnieSharpe · Aug 8, 2009

What is the timeline for getting VX-770 compound into patients' hands?
 
 It is too early to speculate on this. It takes several years for a promising drug to make it from trials to the pharmacy shelf. We are going as fast as we can, but additional trials are necessary before we know whether this promising compound will be approved by the Food and Drug Administration (FDA).
 
 **Taken directly from the CFF website:<a target=_blank class=ftalternatingbarlinklarge href="http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/**
">http://www.cff.org/research/Cl...search/FAQs/VX-770/**
</a> 
 Ronnie

saveferris2009 · Aug 8, 2009

Ronnie is right - drug development is incredibly uncertain and it's nearly impossible to predict timelines.

On average it takes 15-20 years to get a new drug to market.

This drug has FDA "organ drug" status meaning it will get priority review from the FDA. Meaning once ALL trials are finished (and of course assuming trials show positive results), the FDA may take 9 months to review the data instead of a year. Or maybe maybe maybe 6 months.

But look at what happened with aztreonam.... ANYTHING is possible.

We could find out in Phase III trials that VX-770 works great, but there are some side effects we never anticipated - and the FDA could give it the axe.

I'm not taking away anyone's hope - I'm stating facts. Don't shoot the messenger - I didn't make up the facts.

This is why drugs are so expensive - SO MANY fail. Once you get one that works, you must recover the cost of all the other failures (again don't shoot the messenger... this is just a fact).

Take care.

saveferris2009 · Aug 8, 2009

Ronnie is right - drug development is incredibly uncertain and it's nearly impossible to predict timelines.

On average it takes 15-20 years to get a new drug to market.

This drug has FDA "organ drug" status meaning it will get priority review from the FDA. Meaning once ALL trials are finished (and of course assuming trials show positive results), the FDA may take 9 months to review the data instead of a year. Or maybe maybe maybe 6 months.

But look at what happened with aztreonam.... ANYTHING is possible.

We could find out in Phase III trials that VX-770 works great, but there are some side effects we never anticipated - and the FDA could give it the axe.

I'm not taking away anyone's hope - I'm stating facts. Don't shoot the messenger - I didn't make up the facts.

This is why drugs are so expensive - SO MANY fail. Once you get one that works, you must recover the cost of all the other failures (again don't shoot the messenger... this is just a fact).

Take care.

saveferris2009 · Aug 8, 2009

Ronnie is right - drug development is incredibly uncertain and it's nearly impossible to predict timelines.

On average it takes 15-20 years to get a new drug to market.

This drug has FDA "organ drug" status meaning it will get priority review from the FDA. Meaning once ALL trials are finished (and of course assuming trials show positive results), the FDA may take 9 months to review the data instead of a year. Or maybe maybe maybe 6 months.

But look at what happened with aztreonam.... ANYTHING is possible.

We could find out in Phase III trials that VX-770 works great, but there are some side effects we never anticipated - and the FDA could give it the axe.

I'm not taking away anyone's hope - I'm stating facts. Don't shoot the messenger - I didn't make up the facts.

This is why drugs are so expensive - SO MANY fail. Once you get one that works, you must recover the cost of all the other failures (again don't shoot the messenger... this is just a fact).

Take care.

saveferris2009 · Aug 8, 2009

Ronnie is right - drug development is incredibly uncertain and it's nearly impossible to predict timelines.

On average it takes 15-20 years to get a new drug to market.

This drug has FDA "organ drug" status meaning it will get priority review from the FDA. Meaning once ALL trials are finished (and of course assuming trials show positive results), the FDA may take 9 months to review the data instead of a year. Or maybe maybe maybe 6 months.

But look at what happened with aztreonam.... ANYTHING is possible.

We could find out in Phase III trials that VX-770 works great, but there are some side effects we never anticipated - and the FDA could give it the axe.

I'm not taking away anyone's hope - I'm stating facts. Don't shoot the messenger - I didn't make up the facts.

This is why drugs are so expensive - SO MANY fail. Once you get one that works, you must recover the cost of all the other failures (again don't shoot the messenger... this is just a fact).

Take care.

saveferris2009 · Aug 8, 2009

Ronnie is right - drug development is incredibly uncertain and it's nearly impossible to predict timelines.
 
 On average it takes 15-20 years to get a new drug to market.
 
 This drug has FDA "organ drug" status meaning it will get priority review from the FDA. Meaning once ALL trials are finished (and of course assuming trials show positive results), the FDA may take 9 months to review the data instead of a year. Or maybe maybe maybe 6 months.
 
 But look at what happened with aztreonam.... ANYTHING is possible.
 
 We could find out in Phase III trials that VX-770 works great, but there are some side effects we never anticipated - and the FDA could give it the axe.
 
 I'm not taking away anyone's hope - I'm stating facts. Don't shoot the messenger - I didn't make up the facts.
 
 This is why drugs are so expensive - SO MANY fail. Once you get one that works, you must recover the cost of all the other failures (again don't shoot the messenger... this is just a fact).
 
 Take care.

Jeana · Aug 8, 2009