With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.
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The U.S. Food and Drug Administration approved Kalydeco® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.
I have learned a lot about myself in the three years since I started Trikafta®. Even though I am healthier, I feel less connected to the cystic fibrosis community and my care team.
If you receive financial assistance from Vertex GPS™ for a modulator therapy (e.g., Trikafta®, Symdeko®, Orkambi®, or Kalydeco®), you may be impacted by changes Vertex has made to its copay assistance program. The following tips can help you understand how you may be impacted and what next steps may be available to you.
The excitement we experienced anticipating our daughter, Tegan, starting Trikafta® gave way to sadness and confusion as she experienced emotional side effects.
On the brink of being listed for lung transplant, I started Trikafta®, which has helped stabilize my health and helped me avoid it for now, but a transplant could be in my future.
After decades of identifying myself as Monique, “the girl with cystic fibrosis,” I didn’t know who I was anymore after Trikafta changed my health for the better.
Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.