I am sure most of the CF community has heard by now, but Vertex's latest medication to treat the underlying cause of Cystic Fibrosis has completed two phase 3 studies and have met their primary endpoints! First and foremost, I am so excited to see the development of new medications and I am especially excited to see that it benefits my mutation (DF508) as well as other mutations. It gives me so much hope to think in the next year (or so) a newer and better drug than Orkambi, that has helped me with stability, will be available. I am especially hopeful this medication will help me raise my lung function, even if only a little.
The other part of me remembers how I felt when Kalydeco became available. I was excited to see advances in CF care. At the same time I felt heartbroken that Kalydeco was not available to me. It came out at an absolutely horrendous time in my health and hearing story after story of this miracle drug that was changing people's lives was painful when I was feeling as if my life was slipping away. I know so many people with CF are feeling those same mixed feelings with this newest Vertex medication. I also know there are many in the world tonight that have the right mutations, but are still fighting for Orkambi so this medication must also feel out of reach.
I do have hope that all of us with CF, regardless of mutation, will have access to drugs that will completely change the course of this disease. I really hope to see this in my own lifetime.
If you want more details you can find the press release here.