Vertex Pharmaceuticals Inc. has submitted three supplemental New Drug Applications to the U.S. Food and Drug Administration (FDA) to expand eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.
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A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 study results for Trikafta® (elexacaftor/tezacaftor/ivacaftor) for people with cystic fibrosis ages 12 years and older who have one copy of the F508del mutation and one gating or residual function mutation.
The Cystic Fibrosis Foundation recently signed on to a letter with more than 70 organizations in opposition to the Right to Try Act, which passed in the House of Representatives earlier this month.
The Cystic Fibrosis Foundation's successful business model was at the center of a congressional briefing in Washington, D.C., today, which focused on strategies for jump-starting drug development for rare diseases.