rodatristat

Rodatristat is a **potent, peripheral inhibitor of tryptophan hydroxylase 1 (TPH1)**, the rate-limiting enzyme in peripheral serotonin biosynthesis. It is administered as **rodatristat ethyl**, an orally bioavailable prodrug that is converted in the body to active rodatristat. The drug was developed primarily for the treatment of **pulmonary arterial hypertension (PAH)**, aiming to halt or reverse pulmonary vascular remodeling by reducing circulating serotonin. Rodatristat is characterized by its targeted peripheral activity, with limited blood-brain barrier penetration and negligible central nervous system effects. Despite promising preclinical results, clinical trials found that reducing peripheral serotonin with rodatristat ethyl **increased pulmonary vascular resistance and worsened hemodynamic and cardiac function** in PAH patients, leading to termination of further clinical development for this indication[1][2][3][6].

Other names

rodatristat ethyl