The small-molecule specific inhibitor, CFTRinh-172, is the standard reference for the researches. The progresses of CFTRinh-172 in chemical structure, mechanisms of action, and pharmacokinetics were summarizes in this review. CFTRinh-172 plays an important role in the treatment and drug development of malignant diseases such as cholera, polycystic kidney disease and leukemia. Moreover, CFTRinh-172 is the specific molecular inhibitor of the CFTR chloride channel. However, the side-effects and the poor water solubility restrict its application.