FDA approves Promacta for severe aplastic Anaemia - GSK

The FDA has approved a supplemental New Drug Application (sNDA) for the once-daily use of Promacta (eltrombopag), from Glaxo Smith Kline, in patients with severe aplastic Anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST). Approval is based on results from an investigator-sponsored Phase II study (09-H-0154)which demonstrated a haematologic response in SAA patients treated with eltrombopag who had an insufficient response to IST.

In particular, forty per cent of patients experienced a haematologic response in at least one lineage � platelets, red blood cells (RBC), or white blood cells � after Week 12. Ninety-one per cent of patients were platelet transfusion-dependent at baseline; the platelet transfusion-free period in responders ranged from eight to 1,096 days. Eighty-six per cent of patients were RBC-transfusion dependent at baseline; the RBC transfusion-free period in responders ranged from 15 to 1,082 days. The most common adverse reactions in the single-arm, open-label trial, in 43 patients with SAA who received Promacta were nausea (33%), fatigue (28%) and cough (23%).