residual functions and 661

J

jricci

Super Moderator
Excellent news! Thanks so much for sharing. Now that they have more data, hopefully Vertex will resubmit application for a priority review of sNDA for Kalydeco for those with residual function mutations (regardless of whether they have DF508 on other allele) so that patients can gain access asap. And then when the approval for NDA of Tezacaftor/Ivacaftor combination goes through, those with residual function + DF508 would get switched to the combo and those with residual function and not heterzygous for DF508 stay on Kalydeco.
 
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T

triples15

Super Moderator
jricci said:
Excellent news! Thanks so much for sharing. Now that they have more data, hopefully Vertex will resubmit application for a priority review of sNDA for Kalydeco for those with residual function mutations (regardless of whether they have DF508 on other allele) so that patients can gain access asap. And then when the approval for NDA of Tezacaftor/Ivacaftor combination goes through, those with residual function + DF508 would get switched to the combo and those with residual function and not heterzygous for DF508 stay on Kalydeco.
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From your lips to Vertex and the FDA's ears J! Let's hope they move quickly.
 
A

Aboveallislove

Super Moderator
So listened (as best as possible while doing morning treatments with DS and playing Angry Birds Epic with him!

The key for the residual functions: Vertex has ALREADY provided this information to the FDA as part of its prior request to approve Kalydeco for residual functions. And they continue to be in discussions as they have been for the past 18 months. My "gut" is that it will get green lighted soon because of The 21st Century Cures Act OR if not soon, shortly after confirmation of Scott Gottlieb as the FDA Commission. Gottlieb is a big "real world" evidence person who has pushed to give those with serious conditions access sooner, INCLUDING in trials without doing a placebo arm.

The other keys to me were: 1) Better profile, i.e., less adverse events than 770/880 combo; 2) the results were equal to the best of the 770/880 trial. Remember that had 4 arms with different doses in each and the results were all favorable but had a broad range. The 661 combo results for FEV and fewer excerbations equal to the best of the 4 trials; 3) it proved 661 is safe for use in the triple, which is key for those who don't have a drug yet; or and 4) residual functions benefits with combo more than Kalydeco so woo hoo for them!!
 
J

jricci

Super Moderator
Aboveallislove said:
So listened (as best as possible while doing morning treatments with DS and playing Angry Birds Epic with him!

The key for the residual functions: Vertex has ALREADY provided this information to the FDA as part of its prior request to approve Kalydeco for residual functions. And they continue to be in discussions as they have been for the past 18 months. My "gut" is that it will get green lighted soon because of The 21st Century Cures Act OR if not soon, shortly after confirmation of Scott Gottlieb as the FDA Commission. Gottlieb is a big "real world" evidence person who has pushed to give those with serious conditions access sooner, INCLUDING in trials without doing a placebo arm.

The other keys to me were: 1) Better profile, i.e., less adverse events than 770/880 combo; 2) the results were equal to the best of the 770/880 trial. Remember that had 4 arms with different doses in each and the results were all favorable but had a broad range. The 661 combo results for FEV and fewer excerbations equal to the best of the 4 trials; 3) it proved 661 is safe for use in the triple, which is key for those who don't have a drug yet; or and 4) residual functions benefits with combo more than Kalydeco so woo hoo for them!!
Click to expand...

Thanks for the update! Do you remember if they mentioned how many of the 23 mutations were represented in the study?
Also any mention of addressing the many patients with evidence of residual function that have potential to benefit not represented in these trials?
 
A

Aboveallislove

Super Moderator
No and no...unfortunately, but I'd think whatever the initial filing was for because vertex is building on that and not filing a new one. Also on thinking more I bet FDA does move soon..it couldn't before this was public and now that it is makes no sense to wait!
 
T

triples15

Super Moderator
Aboveallislove said:
So listened (as best as possible while doing morning treatments with DS and playing Angry Birds Epic with him!

The key for the residual functions: Vertex has ALREADY provided this information to the FDA as part of its prior request to approve Kalydeco for residual functions. And they continue to be in discussions as they have been for the past 18 months. My "gut" is that it will get green lighted soon because of The 21st Century Cures Act OR if not soon, shortly after confirmation of Scott Gottlieb as the FDA Commission. Gottlieb is a big "real world" evidence person who has pushed to give those with serious conditions access sooner, INCLUDING in trials without doing a placebo arm.

The other keys to me were: 1) Better profile, i.e., less adverse events than 770/880 combo; 2) the results were equal to the best of the 770/880 trial. Remember that had 4 arms with different doses in each and the results were all favorable but had a broad range. The 661 combo results for FEV and fewer excerbations equal to the best of the 4 trials; 3) it proved 661 is safe for use in the triple, which is key for those who don't have a drug yet; or and 4) residual functions benefits with combo more than Kalydeco so woo hoo for them!!
Click to expand...

Thank you Love! So exciting.
 
P

Patti Rowland

New member
yes, Scott Gottlieb is a venture capitalist with many financial ties to the pharmaceutical companies and he will do whatever it takes to push these drugs through..,
 
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