GlaxoSmithKline announced that the Food and Drug Administration (FDA) has approved a supplemental New Drug Application (sNDA) for the use of Promacta (eltrombopag) in patients with severe aplastic anemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).
The approval was based on data from a Phase 2 study (09-H-0154) that evaluated patients with SAA who have had an insufficient response to at least one prior IST and who had a platelet count <30×10^9/L. Results showed that 40% of patients (n=17) experienced a hematologic response in at least one lineage (platelets, red blood cells, or white blood cells after Week 12. Also, 91% of patients were platelet transfusion-dependent at baseline; the platelet transfusion-free period in responders ranged from 8–1,096 days. Similarly, 86% of patients were red blood cell-transfusion dependent at baseline; the transfusion-free period in responders ranged from 15–1,082 days.
The FDA granted Promacta Breakthrough Therapy designation in January 2014 and a Priority Review designation in April 2014. Promacta, a thrombopoietin receptor agonist, is already indicated for thrombocytopenia due to chronic immune (idiopathic) thrombocytopenic purpura (ITP )in adults who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
For more information call (888) 825-5249 or visit Promacta.com.