EU approves Revolade (eltrombopag) for severe aplastic anaemia- Novartis

Novartis has announced the European Commission has approved Revolade (eltrombopag), for the treatment of adults with severe aplastic anaemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and are unsuitable for hematopoietic stem cell transplant.

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) in the USA. The pivotal study demonstrated a haematologic response (40%) in SAA patients treated with eltrombopag 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, increased transaminase, diarrhoea and headache.

Comment: Revolade is the first approved therapy in the EU for patients with severe aplastic anaemia (SAA) who have not responded to other treatments. Of patients treated with standard of care, up to one-third will not respond and approximately 40% of responders will relapse, causing SAA symptoms to return.