Rodatristat Ethyl – a Peripherally Acting TPH Inhibitor
Rodatristat ethyl is a prodrug of a tryptophan hydroxylase (TPH) inhibitor designed to block peripheral serotonin production. By lowering circulating levels of the hormone, rodatristat ethyl may represent a disease-modifying treatment in diseases characterized by excessive production of serotonin, including pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis.
Rodatristat ethyl’s molecular structure was designed to prevent it from crossing the blood-brain barrier, thereby restricting its effect to peripheral serotonin. In nonclinical studies, rodatristat ethyl did not lower central nervous system (CNS) levels of serotonin.
Our initial focus for rodatristat ethyl is in PAH. Rodatristat ethyl is designed to address the underlying cause of pulmonary arterial hypertension (PAH) and Enzyvant is evaluating the ability to impact exercise capacity in patients.
The U.S. FDA and EU EMA granted rodatristat ethyl orphan status under the Orphan Drug Designation program.
Learn more about the disease we are evaluating rodatristat ethyl in PAH here
Learn more about our scientific approach with rodatristat ethyl here