Acceleron Pharma Inc. a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, announced that the European Commission (EC) has granted orphan designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH).
“We’re thrilled at the European Commission’s decision to grant orphan designation to sotatercept in PAH,” said Habib Dable, President and Chief Executive Officer of Acceleron. “We fully intend to take advantage of the benefits that this and other special statuses—including Orphan Drug and Breakthrough Therapy designations in the United States and PRIME designation in Europe—provide to drug developers as we work with health authorities to deliver this potential new backbone therapy in PAH to patients in need as quickly as possible.”
The EC grants orphan designation to medicines intended to treat, prevent or diagnose a disease of low prevalence (fewer than 5 individuals per 10,000 population) that is life-threatening or chronically debilitating. To encourage the development of such medicines, the designation carries with it certain incentives, including scientific advice and assistance with clinical trial protocols, and the potential for a 10-year period of market exclusivity.
Acceleron is currently advancing a Phase 3 development plan for sotatercept, beginning with the registrational trial known as STELLAR expected to initiate by the end of this year.