European Commission Grants Acceleron’s Sotatercept Orphan Drug Designation
Acceleron Pharma has received an Orphan Drug designation in Europe for sotatercept for treating patients with pulmonary arterial hypertension (PAH).
The Cambridge, Mass, company has previously received Orphan Drug and Breakthrough Therapy designations from the FDA and a PRIME designation from the European Medicines Agency — a priority review mechanism for drugs that treat an unmet clinical need.
Sotatercept, which works by rebalancing the molecular signaling that causes PAH, was shown in a phase 2 trial to improve pulmonary vascular resistance and patients’ ability to walk.
Acceleron says the drug will be evaluated in a phase 3 study set to begin by year’s end.