Yes, I know that 661-108 trial doesn’t include the other ½ of residual mutations. I guess I was being overly optimistic in estimating 18 months since there isn’t even a study in place to address the other ½ of residual function mutations. It’s just inconceivable to me that there is so much evidence supporting use of Kalydeco for those with evidence of residual function, yet it isn’t even presently being tested in ½ of those with residual function. I'm not sure where to start in trying to convince Vertex or CFF of adding arm. Any ideas?
I think the phase 3 also requires that the other mutation be f508del, which, unfortunately, is not the other mutation for my daughter (it is w1282x).
Also, anyone who has one of the mutations that is on the list for sure should be jumping up and down and I am definitely happy for all of them. It is not their fault that the list is limited or that Kalydeco is ridiculously expensive.
I guess we can try off label (probably will wait until the residual function label expansion is actually approved). However, I am currently fighting united healthcare trying to get a pancreatic enzyme for my son (he has the same two mutations, but no lung issues, is pancreatic sufficient, but has recurrent attacks of pancreatitis). He's been on zenpep and his Mayo doctor has prescribed Viokase. UHC is insisting that he try Creon first and only after failure, will they approve the viokase. Failure means more trips to the hospital because of pancreatitis (which is incredibly painful). Economically, it makes far more sense for UHC to approve the Viokase, but they haven't yet. Note that my son's doctor has told everyone involved that based on his years of experience, creon will not function any differntly than Zenpep (they're both coated as compared to Viokase). Still, nothing from UHC. Note also that the cost of Viokase pales in comparison to Kalydeco.
We'll keep forging ahead though....