Posted by Michael Wonder on 04 Jan 2018
Novartis receives FDA breakthrough therapy designation for first-line use in severe aplastic anaemia
4 January 2018 - Regulatory filings of the first-line indication in US and EU expected in 2018.
Novartis today announced that the US FDA has granted breakthrough therapy designation to Promacta (eltrombopag) for use in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anemia (SAA) as a first-line therapy.
Promacta, which is marketed as Revolade in most countries outside the US, is already approved as a second-line therapy in the refractory setting in SAA. Promacta is also approved for adults and children with chronic immune thrombocytopenia (ITP), for patients who are refractory to other treatments.
Novartis' analysis of research conducted by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) showed that over half (52%) 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%.
