DF508 and Kalydeco

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moxie1

Guest
I posted this elsewhere, but was not sure anyone could see it so.....
I had clinic visit on Friday. I asked my doctor about starting Kalydeco (I am homozygous DF508) and he said no. He also said that he doesn't know how others are obtaining it because they are requiring proof of mutation.
I was a little disappointed, but not much. My clinic is starting the combo trial of 770/661 and I really want to do it. If I am already on 770, I assume that they are not going to want me.
This being said, is it bad that all of us DF508ers are rushing to get Kalydeco? I understand trying anything if your health is unstable or pfts are low, but for those that are in the higher bracket, maybe we should be waiting so that we are able to be a part of the combo trials that have a better potential to help us.
Anyway, that's my take on it......I could be totally wrong.
 
M

moxie1

Guest
I posted this elsewhere, but was not sure anyone could see it so.....
I had clinic visit on Friday. I asked my doctor about starting Kalydeco (I am homozygous DF508) and he said no. He also said that he doesn't know how others are obtaining it because they are requiring proof of mutation.
I was a little disappointed, but not much. My clinic is starting the combo trial of 770/661 and I really want to do it. If I am already on 770, I assume that they are not going to want me.
This being said, is it bad that all of us DF508ers are rushing to get Kalydeco? I understand trying anything if your health is unstable or pfts are low, but for those that are in the higher bracket, maybe we should be waiting so that we are able to be a part of the combo trials that have a better potential to help us.
Anyway, that's my take on it......I could be totally wrong.
 
S

saveferris2009

New member
I was just thinking about this.

While you make a good point about future clinical trial testing, I have a feeling (just a hunch.... I don't have any info on this whatsoever) they're going to focus on DDF508 patients for any and all class II studies they do with 770 + another drug.

Have a homozygous group of CF genes removes one more variable from clinical trial testing, making results that much more compelling.

They obviously couldn't do homozygous G551D in the 770 trials because G551D is so rare in and of itself, that a homozygous person would be even more rare. The 2nd mutation, along with many other factors such as permanent lung damage, bacterial colonization, age, lifestyle, modifier genes and 2nd CF mutation could account for the variability in FEV1 change in the 770 study.

That's not to say that there won't be variability in a DDF508 study, but perhaps less so than if they were to just recruit and include any patient with DF508 and any other mutation.

So in summary, I think you bring up a valid point - especially if you go to a center where 770+661 is going to be tested (seems to be mostly east coast based right now) AND you have DDF508. But some don't want to risk it or they & their docs feel they will benefit now.

Thanks for bringing up a great point
 
S

saveferris2009

New member
I was just thinking about this.

While you make a good point about future clinical trial testing, I have a feeling (just a hunch.... I don't have any info on this whatsoever) they're going to focus on DDF508 patients for any and all class II studies they do with 770 + another drug.

Have a homozygous group of CF genes removes one more variable from clinical trial testing, making results that much more compelling.

They obviously couldn't do homozygous G551D in the 770 trials because G551D is so rare in and of itself, that a homozygous person would be even more rare. The 2nd mutation, along with many other factors such as permanent lung damage, bacterial colonization, age, lifestyle, modifier genes and 2nd CF mutation could account for the variability in FEV1 change in the 770 study.

That's not to say that there won't be variability in a DDF508 study, but perhaps less so than if they were to just recruit and include any patient with DF508 and any other mutation.

So in summary, I think you bring up a valid point - especially if you go to a center where 770+661 is going to be tested (seems to be mostly east coast based right now) AND you have DDF508. But some don't want to risk it or they & their docs feel they will benefit now.

Thanks for bringing up a great point
 
J

jessykt

Guest
Saveferris: Vertex DID require proof of my G551D mutation before I was able to have my prescription filled. It had to be faxed from the doctor to the company.
 
J

jessykt

Guest
Saveferris: Vertex DID require proof of my G551D mutation before I was able to have my prescription filled. It had to be faxed from the doctor to the company.
 
H

hammerpocket

Guest
Not sure if this is accurate, but in another thread it was mentioned that Vertex requires proof of G551D in order to qualify for their patient support programs, but just getting a prescription filled does not require any proof.
 
H

hammerpocket

Guest
Not sure if this is accurate, but in another thread it was mentioned that Vertex requires proof of G551D in order to qualify for their patient support programs, but just getting a prescription filled does not require any proof.
 
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sue35

New member
So if I get a prescription and give it to CF Services and they ask for proof, that is only for patient support? Does my doctor just say I am not interested in that?
 
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sue35

New member
So if I get a prescription and give it to CF Services and they ask for proof, that is only for patient support? Does my doctor just say I am not interested in that?
 
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saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jessykt</b></i> Saveferris: Vertex DID require proof of my G551D mutation before I was able to have my prescription filled. It had to be faxed from the doctor to the company.</end quote>

Why did you fax anything to the company?
Just take a prescription to a pharmacy or call it in to your mail order pharmacy. There's no need to get Vertex involved.
 
S

saveferris2009

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>jessykt</b></i> Saveferris: Vertex DID require proof of my G551D mutation before I was able to have my prescription filled. It had to be faxed from the doctor to the company.</end quote>

Why did you fax anything to the company?
Just take a prescription to a pharmacy or call it in to your mail order pharmacy. There's no need to get Vertex involved.
 
J

Jeana

New member
I was on the drug study for VX 770 for those homozygous DF508 and they ended the drug study early, because it was not working for this mutation by itself. Did that information ever get released?
 
J

Jeana

New member
I was on the drug study for VX 770 for those homozygous DF508 and they ended the drug study early, because it was not working for this mutation by itself. Did that information ever get released?
 
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