Acceleron Pharma Inc.'s investigational drug sotatercept received the U.S. orphan drug designation to treat patients with a type of hypertension that affects the lung and heart arteries.
Pulmonary arterial hypertension, or PAH, is a chronic disorder that tightens the small pulmonary arteries and raises blood pressure. In some cases, it can lead to heart failure.
"In preclinical studies, sotatercept has demonstrated an ability to target the underlying mechanisms of PAH, which is a rare disease of high unmet medical need. We believe that if similar effects are seen in a clinical setting, sotatercept has the potential to become an important addition to the standard of care in PAH," Acceleron's vice president of pulmonary medical research, Janethe de Oliveira Pena, said in a press release.
The Cambridge, Mass.-based biopharmaceutical company is evaluating sotatercept in two mid-stage trials and expects to report results from one of the studies during the first quarter of 2020.
The U.S. Food and Drug Administration's orphan-drug program is meant for medicines that can treat rare diseases or disorders that affect fewer than 200,000 people in the U.S. With the designation, Acceleron's sotatercept will be eligible for certain incentives, including tax credits for covering clinical trial expenses, prescription drug user fee waivers and seven-year market exclusivity after approval.
