<div class="FTQUOTE"><begin quote><em>Originally posted by: <strong>sAmmie0607</strong></em>You only have one copy of DF508 and the other CFTR mutation Di507 may have been approved with a list of other mutations that fall under the same "gating" or classified mutation as G551D/class III, by the FDA. Im not sure if you fall under this qualification, however it is rare mutation. The only way to receive Kalydeco, is if you have one copy of these genes and you are over the age of six. Those with Double Df508 are not approved by the FDA to receive Kalydeco and would not be approved by medical assistance for drug coverage. Unless it is approved in the future for combination with another drug such as vx-809 or vx-661. Basically it would be a waste of time and money to take Kalydeco alone for double df508, it does not treat the underlying issues of the mutation.</end quote>
Di507 is a class II mutation like DF508 and is not a gating mutation.
The FDA doesn't allow or disallow medications unless there is a REMS program, which Kalydeco does not have.
We, as CFers, use medications off label all the time. Do you take zithromax every day or every other day? That's off-label.... there is no FDA approval for use to decrease inflammation in CF. In fact, the Prescibing Information from the FDA specifically says that zithromax shouldn't be used in CF.
Do you use colistin? Colistin is FDA approved for IV use only....not nebulization. But lots of CFers use this drug off label to treat their PA.
There are countless other examples. Any doctor can prescribe any medication for any reason (provided there isn't a REMS for the medication), including Kalydeco. No one from the FDA or Vertex is checking your mutation (Vertex may check your mutation if you are going to take money for them to subsidize your co-pay.....). Your insurance may ask for documentation, but that's not the FDA.
We have a handful of people, not just me, that don't have a single gating mutation that have Kalydeco.
I sometimes get the feeling that people on this board don't want to get the medication. They're looking for any excuse why they can't get it. Very bizarre. Why not try? Who are you harming if you try? Some of us tried and we have the medication in our bodies right now.
Now the results we get are up in the air - but I'll keep you posted <img src="i/expressions/face-icon-small-smile.gif" border="0">
According to Vertex: "In recombinant cells VX-770 increased CFTR channel open probability (P(o)) in both the F508del processing mutationand the G551D gating mutation."
<a href="http://www.ncbi.nlm.nih.gov/pubmed/19846789">http://www.ncbi.nlm.nih.gov/pubmed/19846789</a>
From Annals of Human Genetics, 2003, by Rowntree et al,:
"Measurements of Cl- conductase of intestine and respiratory tissues of DF508 homozygote CF patients suggest, in vivo, that at least some DF508 CFTR can reach the plasma membrane"
<a href="http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf">http://www.colorado.edu/MCDB/MCDB4600/1ReviewPhenotype.pdf</a>
