Novartis: FDA Approves Promacta For First-Line SAA

(RTTNews) - Novartis announced that the US Food and Drug Administration (FDA) has approved Promacta (eltrombopag) for first-line treatment of severe aplastic anemia (SAA) and Breakthrough Therapy designation for low platelet counts in people exposed to radiation.

Promacta, 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 immunosuppressive therapy (IST). It is also 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.

The FDA's approval is based on Novartis' analysis of research sponsored by the National Heart, Lung and Blood Institute (NHLBI) Division of Intramural Research Program and conducted under a Cooperative Research and Development Agreement (CRADA). The study showed that 44% (95% CI 33, 55) of definitive IST-naive SAA patients achieved complete response at 6 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[1]. The overall response rate was 79% (95% CI 69, 87) at 6 months[2].

Novartis submitted a Type II variation application for Revolade as a first-line SAA treatment to the European Medicines Agency in April 2018 and is expecting a decision in 2019.