Epoprostenol (DB01240): A Comprehensive Pharmacological and Clinical Monograph

1.0 Introduction and Drug Identification

1.1 Overview of Epoprostenol as a Therapeutic Agent

Epoprostenol is a synthetic preparation of prostacyclin (PGI2​), a naturally occurring prostaglandin that functions as a potent vasodilator and a powerful inhibitor of platelet aggregation.[1] Identified as a small molecule drug with the DrugBank accession number DB01240, it has become a cornerstone in the management of severe cardiovascular disease, particularly pulmonary arterial hypertension (PAH).[1]

The therapeutic importance of Epoprostenol is rooted in its landmark regulatory approval. In 1995, it became the first therapy sanctioned by the U.S. Food and Drug Administration (FDA) for the treatment of PAH, a rare and progressive disease.[5] Prior to its introduction, PAH was characterized by a grim prognosis, with a median survival of only 2.8 years from diagnosis.[8] The approval of Epoprostenol, supported by compelling clinical trial data and patient testimony, fundamentally transformed the natural history of the disease, converting it from a rapidly fatal condition into a chronic, manageable illness.[7] This milestone not only provided a life-saving treatment but also validated the prostacyclin pathway as a critical therapeutic target. The success of Epoprostenol spurred the development of an entire class of prostacyclin analogs and related therapies, profoundly altering the therapeutic landscape for patients with pulmonary hypertension.[8]

1.2 Chemical and Physical Properties

Epoprostenol is a complex eicosanoid with a well-defined chemical structure and distinct physical properties. It is supplied commercially as a sterile, white to off-white lyophilized powder that requires reconstitution prior to intravenous administration.[2] The key chemical and identifying properties of Epoprostenol are summarized in Table 1.