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Balovaptan is a selective, orally administered small molecule antagonist of the vasopressin 1A (V1a) receptor. Developed by Roche, it was investigated for the treatment of autism spectrum disorder (ASD), post-traumatic stress disorder (PTSD), and stroke. Balovaptan acts by reversibly binding to V1a receptors in the central nervous system, antagonizing vasopressin signaling and thereby modulating social behaviors and communication. The drug received Breakthrough Therapy Designation from the US FDA for ASD based on early clinical results but failed to show efficacy in later-stage trials for ASD and PTSD. Development has been discontinued for these indications[1][4][5][7].
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