New drug--Kalydeco--Approved

[SIcklyhatED]

My lovely pharmacist sister, who's always researching and keeping us up to date on CF med stuff, just sent me this email about the new drug Kalydeco, which targets the G551D CF mutation. It was just approved by the FDA a few hours ago I believe, and this could lead to the approval/release of a drug to target F508
Here's a link to the article:
<a href="http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm">http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm</a>
They're making progress!

 

[SIcklyhatED]

My lovely pharmacist sister, who's always researching and keeping us up to date on CF med stuff, just sent me this email about the new drug Kalydeco, which targets the G551D CF mutation. It was just approved by the FDA a few hours ago I believe, and this could lead to the approval/release of a drug to target F508
Here's a link to the article:
<a href="http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm">http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm</a>
They're making progress!

 

[saveferris2009]

Yup we already have a thread going on this <img src="i/expressions/face-icon-small-smile.gif" border="0">

http://forums.cysticfibrosis.com/messageview.cfm?catid=5&threadid=615162&enterthread=y

 

[saveferris2009]

Yup we already have a thread going on this <img src="i/expressions/face-icon-small-smile.gif" border="0">

http://forums.cysticfibrosis.com/messageview.cfm?catid=5&threadid=615162&enterthread=y

 

[SIcklyhatED]

hahhaa, my bad. I just finished reading the email was like, "Ahhhh!! MUST. POST. NOW!!." <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[SIcklyhatED]

hahhaa, my bad. I just finished reading the email was like, "Ahhhh!! MUST. POST. NOW!!." <img src="i/expressions/face-icon-small-smile.gif" border="0">

 

[rmotion]

I wonder if they will off label it for use with DDf08?
It did show some improvement just not enough to get a drug passed through FDA
it did have a 6-8% benefit, any thoguths

 

[rmotion]

I wonder if they will off label it for use with DDf08?
It did show some improvement just not enough to get a drug passed through FDA
it did have a 6-8% benefit, any thoguths

 

[Incomudrox]

I would direct everything to the other thread... that was linked above you. However to answer your question I don't see that happening with a drug that cost approx $24k a month....

 

[Incomudrox]

I would direct everything to the other thread... that was linked above you. However to answer your question I don't see that happening with a drug that cost approx $24k a month....

 

[saveferris2009]

maybe ennio can combine the threads?

 

[saveferris2009]

maybe ennio can combine the threads?

 

[Incomudrox]

Doubtful I don't think the software allows for it. Come to think of it, I don't know any forum software that can. It could be done through database editing but it's entirely too much work. If it was me I'd close/lock this one. However it's not me.

 

[Incomudrox]

Doubtful I don't think the software allows for it. Come to think of it, I don't know any forum software that can. It could be done through database editing but it's entirely too much work. If it was me I'd close/lock this one. However it's not me.

 

[saveferris2009]

ya you're right. they can't even get URL's to hyperlink automatically.....

 

[saveferris2009]

ya you're right. they can't even get URL's to hyperlink automatically.....

 

[Anomie]

I think the delta f508's are gonna need all 3 vertex drugs costing 300K a year each. That director at our center told us they need vx 661 and 809 to properly fold the protein and I haven't heard jack about the 661 on the vertex or cff website. Its not even listed as a drug candidate. Maybe I should call them to see what the hold up is.

 

[Anomie]

I think the delta f508's are gonna need all 3 vertex drugs costing 300K a year each. That director at our center told us they need vx 661 and 809 to properly fold the protein and I haven't heard jack about the 661 on the vertex or cff website. Its not even listed as a drug candidate. Maybe I should call them to see what the hold up is.

 

[Incomudrox]

VX-661 is on Vertex's site they (according to Vertex) started phase 2 towards the end of 2011. <div class="FTQUOTE"><begin quote>"The collaboration will help support the development of VX-661. Vertex intends to begin a Phase 2 study of VX-661 by the end of 2011 and expects the study to enroll people with CF who have the F508del mutation. The collaboration will also help support the accelerated discovery and early development of next-generation correctors that aim to treat the underlying cause of CF in people with the F508del mutation. The majority of Vertex's CF research is conducted at Vertex's San Diego, CA research site, where each of Vertex's medicines in development for CF were discovered. Development activities for Vertex's CF medicines are being led by Vertex's research and development site in Cambridge, MA."</end quote>

 

[Incomudrox]

VX-661 is on Vertex's site they (according to Vertex) started phase 2 towards the end of 2011. <div class="FTQUOTE"><begin quote>"The collaboration will help support the development of VX-661. Vertex intends to begin a Phase 2 study of VX-661 by the end of 2011 and expects the study to enroll people with CF who have the F508del mutation. The collaboration will also help support the accelerated discovery and early development of next-generation correctors that aim to treat the underlying cause of CF in people with the F508del mutation. The majority of Vertex's CF research is conducted at Vertex's San Diego, CA research site, where each of Vertex's medicines in development for CF were discovered. Development activities for Vertex's CF medicines are being led by Vertex's research and development site in Cambridge, MA."</end quote>