Tezacaftor/ivacaftor (Symdeko™) is approved for individuals with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 26 specified mutations -- regardless of their other mutation.
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Celtaxsys Inc. reported promising results today from an early stage clinical trial of a potential anti-inflammatory drug for people with cystic fibrosis.
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.
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 the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
When my 8-year-old daughter with CF found out that the disease is potentially fatal, I was grateful for her optimistic views on life and death. But deep down, I was saddened with the thought of a cure being so far away that I wouldn't be around to see it. Today, my fears have dwindled.
Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.