873054-44-5

Product Details:

CasNo： 873054-44-5

Molecular Formula： C24H28N2O3

Appearance： White to off-white Powder

Packing： Aluminium bag

Throughput： 100KG/Month

Purity： 99%

Ivacaftor is a first-in-class cystic fibrosis transmembrane conductance regulator (CFTR) potentiator discovered and developed by Vertex Pharmaceuticals. It is indicated to treat cystic fibrosis (CF) in eligible patients with specific gain-of-function mutations in the CFTR gene.

Unlike CFTR correctors such as tezacaftor that improve protein folding and trafficking, ivacaftor’s mechanism of action targets CFTR channels already present on the cell surface. It increases the open probability of dysfunctional CFTR chloride channels, restoring chloride and bicarbonate transport across epithelial cell membranes. This correction of defective ion transport relieves the hallmark manifestations of cystic fibrosis, including thick, sticky mucus accumulation in the respiratory, gastrointestinal and reproductive tracts.

Ivacaftor can be administered as monotherapy for patients carrying gating mutations such as G551D. It is also a core component of combination regimens, including the tezacaftor/ivacaftor dual therapy and the elexacaftor/tezacaftor/ivacaftor triple-combination therapy. These fixed-dose combinations expand the patient population to include individuals with the prevalent F508del CFTR mutation, greatly broadening access to targeted CF treatment.

Clinically, ivacaftor-based therapies deliver meaningful improvements in lung function, reduce the frequency of pulmonary exacerbations, lower sweat chloride levels and enhance quality of life for cystic fibrosis patients.

Disclaimer: Ivacaftor is an active pharmaceutical ingredient for approved cystic fibrosis medicines. All clinical use of the finished dosage form must comply with local regulatory prescribing information.