Novartis’ Revolade has become the first approved therapy for patients with the rare blood disorder severe aplastic anaemia, after the European Commission green-lighted its use for those who cannot undergo or are failing to respond to standard treatment. 

In SAA the bone marrow does not make enough red blood cells, white blood cells and platelets, leaving patients at risk from serious, life-threatening infections or bleeding. Two out of every one million people in Europe are diagnosed with the condition every year.

Treatment is focused on increasing the number of healthy cells in the blood through IST or haematopoietic stem cell transplantation. But up to a third of patients fail to respond to IST and 30%-40% of responders will relapse. And further highlighting the need for alternative options, around 40% who don't respond die from infection or bleeding within five years of their diagnosis.

Approval came on the back of Phase II data showing a haematologic response (40%) in SAA patients treated with Revolade (eltrombopag) who had an insufficient response to IST. Safety targets were also met, the most common adverse reactions reported in the trial (>=20%) being nausea (33%), fatigue (28%), cough (23%), diarrhoea (21%), and headache (21%).

The drug is already on the US market under the trade name Promacta, for once-daily use in patients with SAA who have had an insufficient response to IST, and for the treatment of children with chronic ITP who have failed to respond to corticosteroids, immunoglobulins or splenectomy.

Elsewhere, Revolade is approved in more than 100 countries for thrombocytopenia in adult patients with chronic ITP, and in over 45 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C, to allow them to initiate and maintain interferon-based therapy.