Regulatory Affairs

Novartis’ Promacta gets FDA breakthrough designation for severe aplastic anemia

PBR Staff Writer Published 05 January 2018

Novartis’ drug Promacta (eltrombopag) has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for first-line treatment of severe aplastic anemia (SAA).

Promacta can now be used in combination with standard immunosuppressive therapy to treat the rare blood disorder.

SAA is a condition in which the bone marrow of the patient cannot produce sufficient red blood cells, white blood cells and platelets.

SAA patients may face debilitating symptoms and complications like fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can restrict their daily activities.

Promacta’s breakthrough designation is backed by data collected from a research by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH).

In the research, it was found that more than half of SAA patients, who were new to any treatment for their condition, achieved complete response at six months to the drug when given with standard immunosuppressive therapy. The overall response rate in study was found to be 85%.

Novartis oncology global drug development head Samit Hirawat said: “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.

“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.”

An oral thrombopoietin (TPO) receptor agonist, Promacta works by inducing stimulation and differentiation of megakaryocytes from bone marrow stem cells to boost production of platelets.

The drug, intended to be taken orally daily, is approved as a second-line treatment in the refractory setting in SAA. It is sold under the brand name Revolade in most countries outside the US.

Promacta is also approved to treat chronic immune thrombocytopenia for patients who do not respond to other treatments.

In 2018, Novartis expects to make regulatory filings of the first-line indication of the drug in the US and the European Union.

Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.