"Today's approval from the European Commission is important news for adults in the EU with severe aplastic anemia, who now have an alternative to standard therapies that have not provided sufficient benefit," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "Revolade helps address an unmet need in this community and underscores our commitment to patients affected by rare diseases."
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 approval is based on the results of a pivotal open-label Phase II study (ELT112523) and two supporting Phase II studies (ELT116826 and ELT116643) conducted by the National Heart, Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH). The pivotal study demonstrated a hematologic response (40%) in SAA patients treated with Revolade who had an insufficient response to IST. The most common adverse reactions (>=20%) in the pivotal single-arm study of 43 patients were nausea, fatigue, cough, transaminase increased, diarrhea, and headache.
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.