Oxytocin (DB00107): A Comprehensive Monograph on its Pharmacological, Clinical, and Neuroscientific Profile

Executive Summary

Oxytocin is a cyclic nonapeptide hormone and neuromodulator with a dual identity that defines its clinical and investigational landscape. As a peripherally acting hormone, it is an indispensable and potent uterotonic agent, classified as a biotech drug and protein-based therapy. Its primary, FDA-approved clinical applications are in obstetrics, where it is used to induce or augment labor, manage inevitable or incomplete abortions, and, most critically, to control and prevent postpartum hemorrhage (PPH). The administration of oxytocin in this context is characterized by a narrow therapeutic index and significant risks, including uterine hyperstimulation, fetal distress, and maternal water intoxication. Consequently, its use demands strict adherence to clinical protocols, including administration via calibrated infusion pump and continuous maternal and fetal monitoring.

Centrally, oxytocin functions as a neurotransmitter within the brain, modulating a complex array of social and emotional behaviors. Often dubbed the "love hormone," its role is more accurately described as a facilitator of social salience, with effects that are highly dependent on individual and contextual factors. This central activity has spurred a vast field of research investigating its therapeutic potential for neuropsychiatric disorders characterized by social deficits, such as autism spectrum disorder (ASD), anxiety disorders, and depression. Despite decades of study, the evidence for these applications remains largely inconclusive and often contradictory. Clinical trials have been hampered by inconsistent findings, methodological challenges related to drug delivery to the brain, and a lack of standardized assays for measuring endogenous oxytocin.