Ivacaftor

Cystic fibrosis, as a rare genetic disease, has long lacked effective treatment methods due to its complex pathogenic mechanism, and this dilemma was not broken until the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Ivacaftor is the first CFTR potentiator drug approved worldwide. It targets the gating defect of CFTR protein to improve the chloride ion transport capacity of CFTR protein with normal function on the cell membrane, thereby alleviating symptoms in multiple organs such as the lungs and digestive system of patients. This drug can be used as a monotherapy for cystic fibrosis patients aged 6 months and older carrying specific CFTR mutations such as G551D, and can also be formulated into compound preparations with other CFTR modulators such as lumacaftor and tezacaftor to cover patient groups with more mutation types, and it is a core component of the current basic treatment for cystic fibrosis.

The global cystic fibrosis treatment market has maintained steady growth in recent years, among which CFTR modulators account for more than 90% of the overall market size. In 2023, the global sales of ivacaftor-related monotherapy and compound products exceeded 8.7 billion US dollars, with a compound annual growth rate maintained at around 12%. At present, the competitive landscape in this field is highly concentrated, and the original research products occupy the vast majority of the global market share. The Chinese market is still in the introduction period, and all approved relevant products are imported, with no generic drugs on the market yet. With the gradual improvement of domestic rare disease security policies, there is a large room for the release of clinical demand for ivacaftor.

The original research enterprise of ivacaftor is Vertex Pharmaceuticals, and the original research trade name is Kalydeco. Its core compound patent in the United States will expire in 2027, and the European compound patent is expected to expire in 2026. The marketed dosage forms of the original research include tablets and oral granules, with specifications covering multiple doses such as 12.5mg, 50mg, 100mg and 150mg, which are convenient for patients of different age groups. The original research dosage form of this product has been included in the FDA Reference Listed Drug Catalog. There is no domestic ivacaftor preparation approved for marketing in China, and the CDE API registration platform has not shown valid and effective registration information of domestic or imported ivacaftor APIs for the time being. (Data as of July 2025, please refer to the official CDE website for the latest information)

CATO can provide a full set of ivacaftor impurity reference standards, most of which are in stock. For in-stock products, orders placed before 16:00 will be shipped on the same day. The products fully meet the compliance requirements of multiple regulations such as the Chinese Pharmacopoeia and FDA, and can provide stable and reliable reference standard support for pharmaceutical enterprises in API R&D, quality research and generic drug consistency evaluation.