Monday, January 12, 2015

Big News From Vertex

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!

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