Elexacaftor

Elexacaftor

Elexacaftor is a medication used in combination with ivacaftor and tezacaftor for the treatment of cystic fibrosis in people aged six years and older who have at least one F508del mutation in the CFTR gene. It is sold under the brand name Trikafta by Vertex Pharmaceuticals.

Mechanism of Action

Elexacaftor works by increasing the amount of functional CFTR protein on the cell surface. This protein helps balance the amount of salt and water on the surfaces of the body, such as the lungs and pancreas. By increasing the amount of functional CFTR protein, elexacaftor helps improve the flow of salt and water in and out of the cells in the lungs and other organs.

Clinical Trials

Elexacaftor was approved by the Food and Drug Administration (FDA) in 2019 based on the results of two clinical trials. The first trial involved 403 patients and the second trial involved 107 patients. Both trials showed that patients who received elexacaftor in combination with ivacaftor and tezacaftor had significant improvements in lung function compared to those who received placebo.

Side Effects

Common side effects of elexacaftor include headache, diarrhea, and upper respiratory tract infection. Serious side effects can include liver problems and cataracts in children and adolescents.

See Also

• Cystic fibrosis
• CFTR protein
• Ivacaftor
• Tezacaftor
• Vertex Pharmaceuticals

References

• FDA approves new breakthrough therapy for cystic fibrosis. FDA News Release. October 21, 2019.
• Vertex Pharmaceuticals Incorporated. TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) tablets, for oral use. U.S. FDA Approved Drug Products. October 21, 2019.