Beraprost: A Comprehensive Pharmacological and Clinical Monograph

Executive Summary

Beraprost is a synthetic, orally active small molecule drug and a pioneering analogue of prostacyclin ().[1] Its primary mechanism of action is as a potent agonist of the prostacyclin (IP) receptor, initiating an intracellular signaling cascade that results in significant vasodilation and potent inhibition of platelet aggregation.[3] This pharmacological profile has established its therapeutic role in several Asian countries, most notably Japan and South Korea, where it is approved for the treatment of peripheral arterial disease (PAD) and pulmonary arterial hypertension (PAH).[1] For patients with PAD, it offers symptomatic relief from ulcers, pain, and coldness associated with chronic arterial occlusion.[5]

Despite its long-standing use in Asia, Beraprost has faced significant challenges in securing global market access. A critical finding from pivotal clinical trials in PAH conducted in Western populations revealed a pattern of initial efficacy, with improvements in exercise capacity and disease progression observed within the first three to six months of therapy, followed by a notable attenuation of these benefits over longer-term treatment.[6] This lack of sustained efficacy, likely linked to the drug's short pharmacokinetic half-life, proved to be a major obstacle for regulatory approval. Consequently, despite being granted orphan drug designation for PAH in both the United States and Europe, these designations were ultimately withdrawn, and the drug has not received marketing authorization from the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA).[8]