Let me start by saying how helpful I’ve found the cysticfibrosis.com website. It has provided a place where I can read how others cope with experiences and situations similar to those I go through. Hopefully some of my comments have also been of some use to them.
The real purpose of this contact is to inform you about my experiences with Kalydeco. My CF doctor recently prescribed it for me “Off Label”. So far Kalydeco is only FDA approved for 9 Class 3 mutations.)
My doctor has another patient doing very well on Kalydeco with similar mutations to mine. (There were some issues getting him the drug.)
When Kalydeco was recently approved for eight additional mutations, my doctor thought it might get approved for me, maybe even as a result of some confusion with the new additions. We were both quite pleasantly surprised when it was approved in less than 24 hours.
Within less than a day after starting Kalydeco I saw significant changes. I started coughing up much more “crap” than I can ever remember. It also seemed thinner than usual. After several days my cough disappeared. It’s hard to remember the last time this happened. The little I was able to cough up after using hypertonic saline was clear. This has happened only once before, about 20 years ago.
It has been several weeks now and in addition to the lack of cough, I’m feeling much better - even though my FEV1 has not significantly improved.
I would have liked to share my experiences with others on the forum. Readers with similar mutations to mine might also be successful in getting Kalydeco “Off Label”.
Perhaps you could inform those with Class 3 and 4 mutations that currently do not have FDA approval, and let them know they might be able to get Kalydeco now. They should contact their doctors and let them know people are getting Kalydeco “Off Label” and are doing very well. They should consider trying to get it too. It might make a tremendous improvement in some or their lives.
My doctor believes that within a year or two, Kalydeco will get FDA approval for all Class 3 and 4 mutations.
The real purpose of this contact is to inform you about my experiences with Kalydeco. My CF doctor recently prescribed it for me “Off Label”. So far Kalydeco is only FDA approved for 9 Class 3 mutations.)
My doctor has another patient doing very well on Kalydeco with similar mutations to mine. (There were some issues getting him the drug.)
When Kalydeco was recently approved for eight additional mutations, my doctor thought it might get approved for me, maybe even as a result of some confusion with the new additions. We were both quite pleasantly surprised when it was approved in less than 24 hours.
Within less than a day after starting Kalydeco I saw significant changes. I started coughing up much more “crap” than I can ever remember. It also seemed thinner than usual. After several days my cough disappeared. It’s hard to remember the last time this happened. The little I was able to cough up after using hypertonic saline was clear. This has happened only once before, about 20 years ago.
It has been several weeks now and in addition to the lack of cough, I’m feeling much better - even though my FEV1 has not significantly improved.
I would have liked to share my experiences with others on the forum. Readers with similar mutations to mine might also be successful in getting Kalydeco “Off Label”.
Perhaps you could inform those with Class 3 and 4 mutations that currently do not have FDA approval, and let them know they might be able to get Kalydeco now. They should contact their doctors and let them know people are getting Kalydeco “Off Label” and are doing very well. They should consider trying to get it too. It might make a tremendous improvement in some or their lives.
My doctor believes that within a year or two, Kalydeco will get FDA approval for all Class 3 and 4 mutations.
