The US Food and Drug Administration has this week accepted Novartis’ supplemental New Drug Application and granted Priority Review for Promacta (in combination with standard immunosuppressive therapy (IST)) for first-line treatment of severe aplastic anaemia (SAA).
Promacta, 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.
Samit Hirawat, Head, Novartis Oncology Global Drug Development, said: “Thanks to the many individuals and organizations who have helped us to advance the development of this promising medicine.
“We will continue our work with the FDA to make Promacta available for this potential new indication as quickly as possible.”
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) and conducted under a Cooperative Research and Development Agreement (CRADA).
The study showed that more than half 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.
Severe aplastic anaemia 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 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.