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Promacta (Eltrombopag) Submitted to FDA for Treatment of Severe Aplastic Anemia
A supplemental new drug application (sNDA) has been submitted to the FDA for Promacta (eltrombopag, GlaxoSmithKline) for the treatment of cytopenias in patients with severe aplastic anemia (SAA) who have shown an insufficient response to immunosuppressive therapy (IST).
SAA is a rare disorder in which the bone marrow fails to produce enough new blood cells. There are no approved therapies for SAA patients who are unresponsive to IST. Of those patients, approximately 40% die from infection or bleeding within 5 years of their diagnosis.
On February 3, 2014, the FDA granted a “breakthrough therapy” designation for eltrombopag in SAA.
The sNDA application was based on positive results from an open-label, phase II National Institute of Health study of eltrombopag in 43 heavily pretreated SAA patients with an insufficient response to IST.
Promacta (eltrombopag) is currently approved in the U.S for the treatment of patients with chronic immune (idiopathic) thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. The drug is not approved for use in SAA.
Source: GlaxoSmithKline; February 28, 2014.