Acceleron Pharma Receives Two FDA Orphan Designations for ACE-536
Published: Mar 26, 2013
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced that the United States Food and Drug Administration (FDA) granted orphan designation for ACE-536 for the treatment of beta-thalassemia and for the treatment of myelodysplastic syndromes (MDS), two rare blood disorders characterized by severe and chronic anemia (low levels of red blood cells). Patients with beta-thalassemia and MDS suffer from ineffective erythropoiesis, a defect in which red blood cell precursors are prevented from maturing into healthy, functional red blood cells resulting in severe anemia. Anemia in these patients is often unresponsive to current therapies and many patients are ultimately dependent on frequent red blood cell transfusions. ACE-536 is an investigational protein therapeutic that increases red blood cells through a novel mechanism and is being developed by Acceleron as part of a global collaboration with Celgene Corporation (NASDAQ: CELG).