Anemoside B4 (AB4), a triterpenoidal saponin derived from Pulsatilla chinensis, has garnered considerable attention for its potent anti-inflammatory and immunomodulatory activities, culminating in its approval for clinical trials by the Center for Drug Evaluation, National Medical Products Administration, for the treatment of mild to moderate ulcerative colitis. Despite this, AB4’s therapeutic potential remained underexplored until the development of its injection formulation. This review discusses the scientific rationale and theoretical framework behind AB4’s development, offering a new paradigm and innovative research strategy for discovering lead compounds or drug candidates from natural medicines. In-depth investigations into AB4’s cellular targets, biochemical pathways, and administration routes have provided valuable insights into its druggability evaluation and clinical potential. The high water solubility of AB4, attributable to its multiple sugar units, imposes limitations on its bioavailability and pharmacokinetic profiles. To address this, structural modification via chemical methods and enzymatic hydrolysis have been employed, resulting in derivatives with reduced molecular weight, improved bioavailability, enhanced pharmacological activity, and greater clinical potential. These advances lay a solid foundation for the continued development of AB4 and its derivatives as promising therapeutic agents.