Novartis announced today that the European Commission has approved Revolade® (eltrombopag) for the treatment of adults with severe aplastic anemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and are unsuitable for hematopoietic stem cell transplant.
SAA is a blood disorder where the bone marrow does not make enough red blood cells, white blood cells and platelets. Two out of every one million people in Europe are diagnosed with aplastic anemia per year, a portion of which are severe cases. The exact cause of the disease is still unknown, but most cases of SAA are believed to be triggered by an autoimmune reaction where the body attacks blood-forming stem cells located in the bone marrow. As a result, patients with SAA are at risk for life-threatening infections or bleeding.
Treatment of SAA is focused on increasing the number of healthy cells in the blood (blood cell count). The current standard of care includes IST or hematopoietic stem cell transplantation. Of patients treated with IST, one-quarter to one-third will not respond and 30-40% of responders will relapse, causing symptoms to return. Approximately 40% of SAA patients who don't respond to initial IST die from infection or bleeding within five years of their diagnosis.
The European Commission approval applies to all 28 EU member states plus Iceland, Norway and Liechtenstein. In August of 2014, eltrombopag (marketed as Promacta® in the USA), was approved by the US Food and Drug Administration for once-daily use in patients with SAA who have had an insufficient response to IST. Eltrombopag is also approved for SAA in Canada.
