Vanzacaftor/tezacaftor/deutivacaftor

Combination drug for cystic fibrosis

Vanzacaftor/tezacaftor/deutivacaftor
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Vanzacaftor/tezacaftor/deutivacaftor is a combination medication used in the treatment of cystic fibrosis (CF), a genetic disorder that affects the lungs and other organs. This medication is designed to improve the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in individuals with cystic fibrosis.

Mechanism of Action

The combination of vanzacaftor, tezacaftor, and deutivacaftor works synergistically to enhance the function of the CFTR protein.

• Vanzacaftor is a CFTR potentiator, which increases the channel opening probability of the CFTR protein, allowing for improved chloride ion transport across cell membranes.
• Tezacaftor acts as a CFTR corrector, aiding in the proper folding and trafficking of the CFTR protein to the cell surface.
• Deutivacaftor is another CFTR potentiator, similar to vanzacaftor, but with modifications that enhance its metabolic stability and duration of action.

Clinical Use

Vanzacaftor/tezacaftor/deutivacaftor is indicated for the treatment of cystic fibrosis in patients who have specific mutations in the CFTR gene. The combination therapy is particularly beneficial for patients with at least one F508del mutation, the most common CF-causing mutation.

Administration

The medication is administered orally, typically in the form of tablets. The dosing regimen is determined based on the patient's age, weight, and specific genetic mutations.

Side Effects

Common side effects of vanzacaftor/tezacaftor/deutivacaftor include headache, dizziness, and gastrointestinal disturbances such as nausea and diarrhea. Patients are monitored for liver function abnormalities, as the medication can affect liver enzymes.

Research and Development

The development of vanzacaftor/tezacaftor/deutivacaftor represents a significant advancement in the treatment of cystic fibrosis, offering improved outcomes for patients with specific genetic profiles. Ongoing research aims to expand the use of this combination therapy to a broader range of CFTR mutations.

Related Pages

• Cystic fibrosis
• CFTR gene
• CFTR modulators