Today's press release from Vertex is causing quite a buzz in the CF community, and that's because there is an anticipated review for acceptance date with the FDA for VX-809 (lumacaftor)! July 5, 2015 is going to be a big day for approximately half of the CF population who carry 2 copies of the F508del mutation!
This is why we, as CF families, work hard to raise money for research, and to help scientists work on finding a cure. These drugs are NOT a cure, and people taking them still have CF, but they're game changing drugs, and will allow CF patients' cells to have some CFTR function, therefore minimizing/slowing down the progress of damage from the disease.
You can read the full press release here.
I have to say that while this is exciting, I can't help but feel bittersweet about it. Some of my friends' children will benefit from this, either now or soon once the studies are completed in the 2-6 age bracket, and I'm happy for them that their moment is coming. But it's a major let down to know that we're still in a holding pattern, waiting to see what the studies show for people like Judith who carry 1 copy of F508del. VX-661 still seems to have better results for these patients, but it still has a way to go in the study process before approval can even be considered.
I know that Judith, and others like her, will have their moment eventually. The waiting is the worst part. But in the meantime, we're going to take a moment to celebrate with the entire community, and rejoice that half of the population will very soon have access to these drugs!