Gentianae Macrophyllae Radix (GMR) is a traditional Chinese herb with the function of dispelling wind and dampness, and removing damp heat. Currently, it is reported in the literature that GMR mainly contains iridoids and secoiridoids, in addition to triterpenoids, flavonoids, lignans, steroids, alkaloids and other chemical components. Among them, iridoids and secoiridoids are the main active ingredients of GMR. Modern pharmacological studies have shown that GMR has pharmacological effects such as anti-inflammatory, anti-tumor, hepatoprotective, immunomodulatory and cardioprotective. In clinical practice, GMR is mainly used to treat rheumatoid arthritis, ulcerative colitis, stroke and other related diseases with good therapeutic effects. Currently, there are few literature reports on the GMR research progress. Therefore, this paper provides a review of the chemical composition, pharmacological activity and clinical applications of GMR to provide some scientific basis for the further development of plant resources.