Novartis has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Promacta (eltrombopag) for use in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anemia (SAA) as a first-line therapy.

Promacta, which is marketed as Revolade in most countries outside the US, is already approved as a second-line therapy in the refractory setting in SAA. Promacta is also approved for adults and children with chronic immune thrombocytopenia (ITP), for patients who are refractory to other treatments.

SAA is a rare blood disorder in which a patient's bone marrow fails to produce enough red blood cells, white blood cells and platelets. As a result, people living with this serious disease may experience debilitating symptoms and complications, such as fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can limit their daily activities. Up to one-third of patients do not respond to current therapies or relapse, causing symptoms to return.  

"Promacta is a promising medicine that, if approved for first-line use in severe aplastic anemia, may redefine the standard of care for patients with this rare and serious bone marrow condition," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development. "We will continue to work closely with the FDA to make Promacta available to patients with SAA who are new to treatment as soon as possible."

Novartis' analysis of research conducted by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) showed that over half (52%) of patients with treatment-naïve SAA achieved complete response at six months when treated with Promacta at the initiation of and concurrently with standard immunosuppressive treatment. The overall response rate was 85%.

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence.

Eltrombopag, marketed as Promacta in the United States and Revolade in countries outside the US, is approved in more than 100 countries worldwide for the treatment of thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an inadequate response or are intolerant to other treatments, approved in over 45 countries worldwide for the treatment of patients with severe aplastic anemia (SAA) who are refractory to other treatments, and also approved in more than 50 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy. Eltrombopag is approved in the US and in the European Union for the treatment of thrombocytopenia in pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids and immunoglobulins.