Pulmonary Arterial Hypertension – A Rare Disease with a High Mortality Rate
Over 250,000 people have been diagnosed with PAH in the US1 and this disease is almost twice as common in women than men.2 PAH is a rapidly progressive disease with an approximate 50% five-year mortality rate.3 The disease is characterized by vascular remodeling in the pulmonary arterial bed. This pathology leads to decreased vessel elasticity, increased arterial blood and resistance in the lungs, and an increased workload for the heart. Individuals with PAH typically succumb to right ventricular heart failure.
PAH is currently treated with vasodilators, which offer some benefit but fail to address the underlying vessel remodeling believed to be responsible for the disease’s high morbidity and mortality rates. Reducing peripheral serotonin production is a novel approach for the treatment of PAH and has the potential to halt or reverse pulmonary remodeling. Rodatristat is being developed as an adjunctive treatment for PAH as its mechanism of action is thought to be complementary to the current standard of care.