The U.S. FDA has granted Orphan Drug Designation to Fostamatinib for the treatment of immune thrombocytopenic purpura (ITP).  Fostamatinib is an oral spleen tyrosine kinase inhibitor under clinical development by Rigel Pharmaceuticals, a biotechnology company based in South San Francisco, CA.  ITP is a rare form of autoimmune disease that the patient’s immune system begins to attack the body’s own platelets resulting in excessive bruising and bleeding.  Current therapeutic options include corticosteroids, blood platelet production boosters and splenectomy.  Rigel believes that fostamatinib may address the autoimmune basis of the disease and is conducting a Phase III clinical trial in patients with ITP.


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