FDA grants expanded approval for Novartis' Promacta in first-line severe aplastic anaemia

Novartis announced that the FDA has expanded the label for Promacta (eltrombopag) to include first-line treatment for adults and paediatric patients two years and older with severe aplastic anaemia (SAA), in combination with standard immunosuppressive therapy (IST). The oral thrombopoietin receptor agonist is already approved for SAA in patients who have had an insufficient response to IST. 

"SAA can be a fatal diagnosis if left untreated, and many patients fail to respond to current initial treatment options," commented Liz Barrett, chief executive of Novartis Oncology. The company noted that "Promacta is the first new treatment in decades for newly diagnosed SAA patients in the US." 

According to Novartis, the latest approval is based on the company's analysis of research sponsored by the National Heart, Lung and Blood Institute division of the US National Institutes of Health's Intramural Research Programme. Findings from the study demonstrated that 44 percent of definitive IST-naïve patients with SAA achieved complete response at six months when treated with Promacta concurrently with standard IST, making it 27 percent higher than the complete response rate historically observed with standard IST alone, the company said. The drugmaker also indicated that the overall response rate at six months for Promacta-treated patients was 79 percent. 

"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," Novartis stated. The company added that the new data also showed a median duration of response of 24.3 months for patients receiving six months of Promacta in combination with horse anti-thymocyte globulin and cyclosporine (CsA) followed by maintenance CsA4. 

Separately, Novartis announced that the FDA also designated Promacta a breakthrough therapy as a counter measure for haematopoietic sub-syndrome in patients suffering from radiation sickness. 

The Swiss drugmaker obtained the therapy through an asset swap with GlaxoSmithKline, which was finalised in 2015. 

Promacta is also approved in the US for adults and children with chronic immune thrombocytopaenia (ITP) who are refractory to other treatments, and for the treatment of thrombocytopaenia in patients with chronic hepatitis C virus infection. 

The therapy is marketed as Revolade in Europe, where it is also approved for adults with SAA who have not responded to other treatments, as well as for splenectomised adults with ITP who have not responded to other treatments. Novartis stated that it has already filed a Type II variation application for Revolade as a first-line SAA treatment to the European Medicines Agency, with a decision expected in 2019. 

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