Yesterday was a crazy day. The FDA Advisory Panel was hearing presentations about why it should recommend approval for a drug that I’ve been waiting for for about 5+ years. I was reading a live blog here and there during the day, but I wanted to wait until I got home to hear the news. I told my mom not to tell me what the decision was… as this was a big moment for me and I wanted to read the news myself.
So I got home, scrolled towards the end of the blog and read. “12 Yes, 1 No. The FDA Advisory Committee has recommended approval for Orkambi.” It was over-wheleming, surreal, happy and scary. It was a moment that has seemed so far off since I heard rumblings about this CFTR potentiator back when I was in high school. It was something that would change the way my cells function and make them function normally. A treatment like this has always given me hope, it’s been the light at the end of the tunnel, it has always been something that would eventually happen.
But, it’s been a roller coaster ride.
First Kalydeco was approved and I heard all of these miracle stories. Then, I would hear that the results for lumacaftor/ivacaftor (for my mutation) weren’t THAT good, investors were disappointed. Then someone would point out that it decreased exacerbations and I’d regain my hope. Then I’d hear that there are all sorts of different potentiators being researched and my hope would come back, but the clock would reset knowing how long these drugs actually take to come to fruition.
And now, it’s in reach. It will still be at least a couple of months until the FDA approves it. But it’s real now. The impact may be smaller than I expected, but the big takeaway from the meeting yesterday is that THIS SLOWS THE PROGRESSION OF THE DISEASE. As I’ve watched my lung function go down significantly these last 5 years, that’s huge. It never seemed real to me, but yesterday everything was put into perspective. This will change everything.
So I cried, and got it all on camera… obviously: https://www.youtube.com/watch?v=xXSf0l8OqKE&feature=youtu.be
