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Good News for Cystic Fibrosis
A remarkable new drug could make a difference for tens of thousands of Americans with CF or cystic fibrosis. It may improve their quality of life and extend it.
Cystic fibrosis is a mutation of the CFTR gene which makes a defective protein that's unable to move chloride from the inside of cells to the outside. This means water is not attracted to the cell surface making the mucus in certain organs very thick and sticky. In lungs, the thick mucus clogs airways and traps bacteria causing repeated infections which can lead to respiratory failure. But the new drug, called Trikafta alleviated symptoms for all patients in two studies.
The key to Trikafta is that it targets the most common mutation in the CFTR gene. This mutation causes the CFTR protein to misfold making it nonfunctional and causes ninety percent of CF cases.
In one drug trial, about four hundred patients took Trikafta or a placebo. The second trial involved just over one hundred patients who took either Trikafta or another established treatment. Both studies measured the maximum amount of air a person can forcibly expel in one second, called the ppFEV1 , to mark CF progression. In both studies, this measure improved by nearly fourteen percent in one group and ten percent in the other. Two patients even ran a five-K race, their symptoms had improved so much.
Trikafta could be transformative by preventing accumulating lung damage so that people can extend their lifespan by decades more.
More Information
FDA approves new breakthrough therapy for cystic fibrosis
Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options...
FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment
FDA Grants Priority Review of the application and sets a PDUFA target action date of March 19, 2020. Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations...
Basics of the CFTR Protein
Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all. By understanding how the protein is made, scientists have been able to develop treatments that target the protein and restore its function...
