FDA review of Promacta® for severe aplastic anemia (SAA)

Novartis have announced that the US Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review designation to Promacta® (eltrombopag) in combination with standard immunosuppressive therapy (IST) for first-line treatment of severe aplastic anemia (SAA).

Promacta, which is marketed as Revolade® in most countries outside the US, is an oral thrombopoietin receptor agonist (TPO-RA) that is already approved for SAA in the refractory setting for patients who have had an insufficient response to IST. It is also approved for adults and children with chronic immune thrombocytopenia (ITP) for patients who are refractory to other treatments and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.

The Priority Review for first-line SAA is based on Novartis’ analysis of research sponsored by the Intramural Research Program of the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH). The study showed that more than half (52%) of treatment-naïve SAA patients achieved complete response at six months when treated with Promacta concurrently with standard IST, which was an increase of 35% compared to those treated with the standard IST alone. The overall response rate was 85% at six months.

Severe aplastic anemia is a rare, life-threatening, acquired 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 may experience fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can limit their daily activities. Historically, SAA was nearly uniformly a fatal diagnosis due to infection or hemorrhage resulting from prolonged pancytopenia; untreated SAA can result in 80-90% mortality in 1-2 years. The prevalence rates vary for aplastic anemia in the US, but it is believed that 500-1,000 new cases are diagnosed each year.

Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development said, “Promacta is a great example of our drive to develop innovative treatments in serious disease areas where few treatment options exist.”