Tezacaftor (VX-661) is a novel small molecule developed by Vertex Pharmaceuticals as a potential therapy for cystic fibrosis (CF). It is intended to be used in combination with another CFTR modulator, ivacaftor, to form a combination therapy known as "tezacaftor/ivacaftor" or "Trikafta". Tezacaftor is a CFTR corrector and is believed to increase the amount of functional CFTR protein at the cell surface. It is an orally administered, small molecule that has been shown to improve the function of the defective CFTR protein in laboratory studies. It is currently in clinical trials for the treatment of CF. The combination of tezacaftor and ivacaftor has been approved by the United States Food and Drug Administration and the European Commission for the treatment of CF in people aged 12 and older who have at least one F508del mutation.
Tezacaftor is a medication used in combination with another drug, ivacaftor, to treat cystic fibrosis (CF) in people six years and older who have a specific genetic mutation called F508del. This mutation is the most common type of mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Cystic fibrosis is an inherited disorder that causes thick, sticky mucus to build up in the lungs, digestive tract, and other organs. This mucus can cause breathing problems, infections, and other serious complications. Tezacaftor works by helping the body to produce more functional CFTR protein. This protein helps to move salt and water in and out of cells. By improving the function of the CFTR protein, tezacaftor can help to thin mucus and improve lung function in people with cystic fibrosis.
Tezacaftor is available as a capsule that is taken by mouth, twice a day. It is important to take tezacaftor with ivacaftor exactly as prescribed by your doctor. Tezacaftor can cause side effects, including headache, diarrhea, nausea, and vomiting. It can also interact with other medications, so it is important to tell your doctor about all the medications you are taking before starting tezacaftor.
Here are some additional details about Tezacaftor:
- Brand Name: Symdeko (in combination with ivacaftor)
- Drug Class: CFTR corrector
- FDA Approved: 2018 (in combination with ivacaftor)
- Available As: Capsule