On December 7, 2015 Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) reported that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to luspatercept for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (MDS) (Press release, Acceleron Pharma, DEC 7, 2015, View Source [SID:1234508446]). The Fast Track program of the FDA is designed to facilitate the development, and expedite the review, of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
Celgene and Acceleron are developing luspatercept to treat patients with rare blood disorders, including MDS and beta-thalassemia. For people with MDS, a type of blood cancer, anemia is a challenging clinical complication of the disease. Patients receive chronic red blood cell transfusions as standard of care to manage the anemia. Luspatercept is designed to increase red blood cell levels in a way that is fundamentally distinct from existing therapies, and has the potential to treat anemia in patients whose disease is not adequately addressed by the treatments available today.
“The FDA’s Fast Track Designation for luspatercept recognizes the serious unmet medical needs of patients with MDS,” said Jacqualyn A. Fouse, President, Hematology/Oncology for Celgene. “Celgene and Acceleron are working to initiate a Phase 3 clinical program to investigate the treatment of patients with low- and intermediate- risk MDS before the end of the year.”
The Companies previously announced FDA Fast Track Designation for luspatercept to treat beta-thalassemia.
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-beta) superfamily involved in the late stages of erythropoiesis (red blood cell production). Luspatercept is thought to regulate late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation. This mechanism of action is distinct from that of erythropoietin (EPO), which stimulates the proliferation of early-stage erythrocyte precursor cells. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Acceleron and Celgene are initiating phase 3 clinical trials that are designed to evaluate the safety and efficacy of luspatercept in patients with myelodysplastic syndromes (the “MEDALIST” study) and in patients with beta-thalassemia (the “BELIEVE” study). For more information, please visit www.clinicaltrials.gov.
About Myelodysplastic Syndromes
MDS comprise a heterogeneous group of hematologic malignancies of the bone marrow commonly leading to severe and chronic anemia due to ineffective erythropoiesis. The National Cancer Institute estimates that more than 10,000 people are diagnosed with MDS in the United States each year. Patients with MDS often have a hypercellular bone marrow with various dysplastic changes of the cells that are also seen in peripheral blood, resulting in cytopenias (low blood cell counts) and an increased risk of progression to acute myeloid leukemia. Nearly all MDS patients suffer from anemia. The anemia in MDS is often characterized by high endogenous levels of EPO driving an abundance of early stage red blood cell precursors and an inability of these precursor cells to properly differentiate into healthy, functional red blood cells. Many patients are therefore unresponsive to the administration of erythropoietin to correct the resulting anemia and instead require red blood cell transfusions, which can increase the risk of infection and iron-overload related toxicities.