GSK‘s treatment for cytopenias in patients with severe aplastic anemia (SAA) who have had insufficient response to immunosuppressive therapy, has been granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration (FDA). This is the FDA’s 32nd Breakthrough Therapy Designation.
Eltrombopag (Promacta, USA; Revolade, Europe) is not yet licensed for use anywhere in the world as a treatment for aplastic anemia. The FDA granted the small molecule thrombopoietin receptor, developed in partnership between GSK and Ligand, breakthrough therapy designation on Monday 3rd February, having previously awarded it Orphan Drug Designation (ODD) in November for Aplastic Anemia, as well as earlier in May 2008 for Idiopathic Thrombocytopenic Purpura (patients with low platelet counts but normal bone marrow and an absence of other causes).
SAA is a rare disorder in which a patient’s bone marrow cannot produce enough new blood cells. For patients who are unresponsive to immunosuppressive therapies there are few options available. Around 40% of patients who are unresponsive to traditional SAA treatments die of infection or bleeding within five years of their diagnosis.
After promising results from a pilot trial, an expansion trial was completed with the following results: 7 patients experienced a complete, trilineage response; 9 patients were transfusion-independent for platelets; 6 patients had improved hemoglobin levels; 3 patients who were previously dependent on red cell transfusions achieved transfusion-independence; and 8 patients exhibited increased neutrophil counts.
See the press release here.
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