Revolade approved by EC to treat patients with severe aplastic anemia, a serious blood disorder
The European Commission ( EC ) has approved Revolade ( Eltrombopag ) 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.
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 severe aplastic anemia 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 severe aplastic anemia are at risk for life-threatening infections or bleeding.
Treatment of severe aplastic anemia is focused on increasing a patient's blood cell count. The current standard of care includes immunosuppressive therapy or hematopoietic stem cell transplantation. Of patients treated with immunosuppressive therapy, one-quarter to one-third will not respond and 30-40% of responders will relapse, causing symptoms to return. Approximately 40% of severe aplastic anemia patients who don't respond to initial immunosuppressive therapy 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 patients with severe aplastic anemia treated with Revolade who had an insufficient response to immunosuppressive therapy.
The most common adverse reactions ( greater than or equal to 20% ) in the pivotal single-arm study of 43 patients were nausea ( 33% ), fatigue ( 28% ), cough ( 23% ), diarrhea ( 21% ), and headache ( 21% ).
In the single-arm, single-center, open-label study, Eltrombopag was evaluated in 43 patients with severe aplastic anemia who have had an insufficient response to at least one prior immunosuppressive therapy and who had a platelet count less than or equal to 30 x 109/L.
At baseline, the median platelet count was 20 x 109/L, hemoglobin was 8.4 g/dL, absolute neutrophil count ( ANC ) was 0.58 x 109/L, and absolute reticulocyte count was 24.3 x 109/L.
The treated population had a median age of 45 years ( range 17 to 77 years ) and 56% were male. The majority of patients ( 84% ) received at least two prior immunosuppressive therapies.
Eltrombopag was administered at an initial dose of 50 mg once daily for two weeks and increased over two-week periods up to a maximum dose of 150 mg once daily.
The primary endpoint was hematologic response, which was initially assessed after 12 weeks of treatment with Eltrombopag. Treatment was discontinued after 16 weeks if no hematologic response was observed. Additional efficacy assessments included median duration of response in months. ( Xagena )
Source: Novartis, 2015
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