Ivacaftor hydrate is a specialty chemical and active pharmaceutical ingredient (API) primarily used in the research and development of treatments for cystic fibrosis. As a synthetic organic compound (molecular formula: C₂₄H₂₈N₂O₃ · H₂O; CAS: 1159836-45-0), it functions as a CFTR potentiator, enhancing the function of the defective CFTR protein in patients with specific gating mutations, such as G551D. This action improves chloride ion transport, leading to better hydration and mucus clearance in the airways. In clinical applications, it targets the underlying cause of cystic fibrosis, improving lung function, reducing pulmonary exacerbations, and enhancing quality of life. It is typically used as long-term maintenance therapy, often in combination with other CFTR modulators, and is suitable for patients aged 6 months and older with responsive CFTR mutations.