Therapy Areas: Respiratory
Novartis wins US FDA approval for Promacta for first-line SAA and announces breakthrough therapy designation for H-ARS
19 November 2018 -

Medicines company Novartis reported on Friday the receipt of approval from the US Food and Drug Administration (FDA) to expand the label for Promacta to include first-line treatment for adults and pediatric patients two years and older with severe aplastic anemia (SAA) in combination with standard immunosuppressive therapy (IST).

SAA 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.

Promacta (eltrombopag), 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 for patients who have had an insufficient response to IST. It is approved for adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments, and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.

This US FDA approval is based on the company's analysis of research sponsored by the National Heart, Lung and Blood Institute (NHLBI) Division of Intramural Research Programme and conducted under a Cooperative Research and Development Agreement (CRADA).

The study showed that 44% of definitive IST-naive SAA patients achieved complete response at six months when treated with Promacta concurrently with standard IST, which was 27% higher than the complete response rate historically observed with the standard IST alone. The overall response rate was 79% at six months.

In April 2018, Novartis submitted a Type II variation application for Revolade as a first-line SAA treatment to the European Medicines Agency and is expecting a decision in 2019. The US FDA granted Promacta Breakthrough Therapy designation as a counter measure for hematopoietic sub-syndrome of acute radiation syndrome (H-ARS).

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