Ivacaftor is a CFTR potentiator used in the treatment of cystic fibrosis in patients with specific mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene.
Unlike conventional antibiotics or anti-inflammatory medicines, ivacaftor directly enhances the activity of certain defective CFTR chloride channels at the cell surface. This improves chloride and fluid transport across epithelial tissues.
Ivacaftor is a specialized small-molecule pharmaceutical API manufactured through controlled chemical synthesis, purification and crystallization.
Critical quality attributes include identity, assay, related substances, water content, residual solvents, elemental impurities, particle characteristics and polymorphic form.
CAS Number: 873054-44-5
Molecular Formula: C24H28N2O3
Molecular Weight: 392.49 g/mol
Drug Class: CFTR Modulator
Pharmacological Class: CFTR Potentiator
Therapeutic Class: Cystic Fibrosis Treatment
Therapeutic Area: Respiratory / Genetic Disorders
Ivacaftor increases the probability that activated CFTR chloride channels remain open at the epithelial cell surface.
This enhances chloride transport and improves epithelial surface hydration.
The resulting effects include:
Ivacaftor is therefore classified as a CFTR potentiator rather than a CFTR corrector.
Ivacaftor is used in patients with cystic fibrosis who have specific responsive CFTR mutations.
It may be used as:
The appropriate indication depends on the patient's mutation, age and the approved regulatory labeling.
Ivacaftor is particularly important in precision treatment of cystic fibrosis because its activity depends on the presence of a CFTR variant responsive to potentiation.
It has also been combined with CFTR correctors such as lumacaftor, tezacaftor and elexacaftor in different therapeutic regimens.
Typical API manufacturing may involve:
Ivacaftor API quality evaluation may include:
Ivacaftor should be stored in a tightly closed container under controlled pharmaceutical storage conditions, protected from excessive heat, moisture and direct light. Final storage conditions should be established using validated stability data.
Ivacaftor is relevant to research involving:
Ivacaftor is a small-molecule CFTR potentiator with CAS Number 873054-44-5, molecular formula C24H28N2O3, and molecular weight 392.49 g/mol. It enhances the opening probability of responsive CFTR chloride channels and is used for mutation-specific treatment of cystic fibrosis.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| Heavy Metals | NMT 20 ppm |
| pH (1% Solution) | 4.0 – 7.0 |
| Individual Impurity | NMT 0.5% |
| Total Impurities | ≤ 1.5% |
| Water Content | NMT 1.0% |