Icenticaftor

Icenticaftor

Icenticaftor is a pharmacological agent that is used in the treatment of cystic fibrosis. It is a type of potentiator that works by increasing the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is often dysfunctional in individuals with cystic fibrosis.

Mechanism of Action

Icenticaftor functions as a CFTR potentiator. The CFTR protein is a channel that transports chloride ions across cell membranes. In individuals with cystic fibrosis, mutations in the CFTR gene lead to the production of a dysfunctional CFTR protein, resulting in the buildup of thick, sticky mucus in various organs. Icenticaftor enhances the function of the CFTR protein, thereby improving the transport of chloride ions and the hydration of mucus.

Clinical Use

Icenticaftor is used in combination with other drugs, such as lumacaftor and tezacaftor, to treat cystic fibrosis in individuals who have specific mutations in the CFTR gene. The combination of these drugs has been shown to improve lung function and reduce the frequency of pulmonary exacerbations in individuals with cystic fibrosis.

Side Effects

Common side effects of icenticaftor include headache, upper respiratory tract infection, abdominal pain, and diarrhea. Serious side effects can include liver problems and cataracts.

Development and Approval

Icenticaftor was developed by Vertex Pharmaceuticals and was approved by the Food and Drug Administration (FDA) in the United States in 2012. It is marketed under the brand name Kalydeco.

See Also

• Cystic fibrosis
• Cystic fibrosis transmembrane conductance regulator
• Lumacaftor
• Tezacaftor
• Vertex Pharmaceuticals