CHMP recommends Revolade (eltrombopag)to treat severe aplastic anemia-Novartis
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Revolade (eltrombopag) from Novartis for the treatment of adult patients with severe aplastic anemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST) and are not eligible to receive a hematopoietic stem cell transplant.
The CHMP positive opinion 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 (33%), fatigue (28%), cough (23%), diarrhea (21%), and headache (21%).
Comment: Severe aplastic anemia is a blood disorder where the bone marrow fails to make enough red blood cells, white blood cells and platelets. Two out of every one million people in Europe and North America 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 a patient's 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 do not respond to initial IST die from infection or bleeding within five years of their diagnosis.
Comment: eltrombopag was approved by the FDA for treatment of severe aplastic anaemia as Promacta in August 2014.
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