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The new triple combo from Vertex
Hi.
I have a few questions that has been bugging me.
1. Is it fair to assume that the triple combination work better on homozygous f508del patients? They do have two mutations susceptible to correction rather than one. I’ve seen the in vitro results that there is indeed greater correction of CFTR, but not in vivo. That leads me to my second question.
2. Given that these P2 trials have proven FEV1 increases about the same for heterozygous and homozygous patients. Does this mean that the “ceiling” has been reached in terms of how much mucus a patient can harbour in their lungs (13-14%)? Do you think these meds have reached a plateau in terms of being able to “getting-the-stuff-that-are-not-supposed-to-be-in-the-lung” out?
If yes to my second question, then the most important goal has been reached in terms of controlling the disease, ie the lung. If patients achieve normal mucus clearance and their cilia can work like they are supposed to, there is a real reason to get excited.
I am beyond enthusiastic for these to be available!
Other metrics like BMI, endocrine and exocrine pancreas functionality etc are also likely to improve.
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