Novartis could soon offer SAA patients new treatment, as it on Thursday announced getting the Breakthrough Therapy for its Promacta combination in the USA from the FDA. It is to be used with standard immunosuppressive therapy for the severe aplastic anemia (SAA) patients, as a first-line therapy.
Promacta, is already approved as a second-line therapy in the refractory setting in SAA in most countries outside the US marketed as Revolade. It is also approved for adults and children with chronic immune thrombocytopenia (ITP), for patients who are refractory to other treatments.
What is SAA?
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. Novartis noted that 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. He added that the company will focus on getting Promacta on drugstore shelves as soon as possible.
Novartis said that its analysis of research conducted by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) showed that over half 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%.
Image: The logo of Swiss drugmaker Novartis is seen at its headquarters in Basel, Switzerland October 22, 2013. REUTERS/Arnd Wiegmann/File Photo