Novartis has announced the publication of data showing high response rates in patients with the rare blood disorder severe aplastic anaemia (SAA) taking its medicine eltrombopag, supporting its first-line use for the condition.

In the study, carried out by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) and published in the New England Journal of Medicine, 58 percent of patients with treatment-naïve SAA achieved complete response at six months when treated with eltrombopag at the initiation of and concurrently with standard immunosuppressive treatment.

In patients with SAA the bone marrow fails to make enough red blood cells, white blood cells and platelets, giving rise to debilitating symptoms and complications, such as fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding.

The current standard of care includes immunosuppressive therapy (IST) or haematopoietic stem cell transplantation, but  up to one-third of patients will not respond to IST and 30-40 percent of responders will relapse, causing symptoms to return, Novartis notes.

"Our research in NEJM shows that eltrombopag plus standard immunosuppressive therapy appeared to increase the overall response rate and substantially increase the frequency, speed and robustness of haematologic recovery in patients with SAA compared to historical controls," noted the study's lead author, Danielle Townsley, MD, researcher in the NHLBI.

"Eltrombopag is the only thrombopoietin receptor agonist to be used in the second-line treatment of SAA, and these results from the NIH study now show its potential as a first-line treatment, which we look forward to discussing with health authorities,” said Vasant Narasimhan, global head of drug development and chief medical officer at Novartis.

The drug, which is marketed as Promacta in the US and Revolade in countries outside the US, has racked up approvals for thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an inadequate response or are intolerant to other treatments, SAA in patients refractory to other treatments, and thrombocytopenia in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy.

In the US and EU, it is also cleared for the treatment of thrombocytopenia in paediatric patients one year and older with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.