GSK has submitted to the European Medicines Agency (EMA) of a variation to the Marketing Authorisation for eltrombopag (Revolade), seeking an additional indication for the treatment of adult patients with severe aplastic anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).

SAA is a rare disorder in which the bone marrow fails to make enough new blood cells. There are currently no approved therapies available in the European Union (EU) for patients with SAA. Of those patients unresponsive to initial IST, approximately 40% die from infection or bleeding within five years of their diagnosis.

The EMA submission is based on the results of a pivotal open-label phase II study (ELT112523) and two supporting phase II studies (ELT116826 and ELT116643) by the National Institute of Health evaluating eltrombopag in patients with SAA.

On 26 August, 2014, eltrombopag—marketed as Promacta in the USA and as Revolade in Europe and other countries across the world—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 not approved or licensed anywhere else in the world for use in SAA.

In addition, eltrombopag is approved in more than 95 countries worldwide for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) and in 43 countries worldwide for the treatment of thrombocytopenia (low blood platelet counts) in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy.

Promacta and Revolade are registered trademarks of the GSK group of companies.