FDA approves expended label for Promacta to include first-line treatment for adults and pediatric patients two years and older with severe aplastic anemia.-Novartis

Novartis announced that the FDA has expanded the label for Promacta (eltrombopag) to include first-line treatment for adults and pediatric patients two years and older with SAA ( severe aplastic anemia)in combination with standard immunosuppressive therapy (IST). 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 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.

"Severe aplastic anemia can be a fatal diagnosis if left untreated, and many patients fail to respond to current initial treatment options," said Liz Barrett, CEO, Novartis Oncology. "Today's US approval for Promacta is an important step forward for people living with this challenging disease and shows how Novartis continues to reimagine care in areas where few treatment options exist."

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. The overall response rate was 79% (95% CI 69, 87) at 6 months.These high response rates have a significant clinical impact for patients with SAA who were not previously treated with standard IST. These results further build on the IST-refractory indication for patients with SAA which Promacta was granted in 2015, in which a subset of patients maintained stable counts and demonstrated restoration of bone marrow function following Promacta discontinuation.

The new data in IST-na�ve SAA includes sustained response with a median duration of response of 24.3 months for patients receiving 6 months of Promacta in combination with horse anti-thymocyte globulin (h-ATG) and cyclosporine (CsA) followed by maintenance CsA4. In this study, the most common adverse reactions reported (incidence greater than =5%) were abnormal liver function tests, rash, and skin discoloration including hyperpigmentation.

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..