Elexacaftor is a small-molecule CFTR modulator and active pharmaceutical ingredient used in the treatment of cystic fibrosis (CF) in combination with other CFTR-modulating medicines. It is classified as a CFTR corrector and is designed to improve the processing and trafficking of certain defective cystic fibrosis transmembrane conductance regulator (CFTR) proteins.
Elexacaftor is used as part of combination therapy, particularly in products containing elexacaftor, tezacaftor and ivacaftor. The combination is designed to address different aspects of defective CFTR protein function.
Elexacaftor has the molecular formula C26H27F2NO4 and a molecular weight of approximately 479.50 g/mol. Its CAS Registry Number is 1353858-99-7.
Elexacaftor API is a pharmaceutical active ingredient used in the development and manufacture of CFTR-modulator pharmaceutical formulations. It is particularly relevant to cystic fibrosis drug-development programs involving CFTR correction and combination therapy.
| 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) | 2.0 – 4.0 |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |
| Melting Point | 150°C – 154°C |