The US Food and Drug Administration (FDA) has accepted Novartis’ supplemental new drug application (sNDA) and granted priority review designation to Promacta (eltrombopag) in combination with standard immunosuppressive therapy (IST) for first-line treatment of severe aplastic anaemia (SAA).
approval
Promacta is an oral thrombopoietin receptor agonist (TPO-RA) that has already been approved for the treatment of SAA in the refractory setting for patients who have had an insufficient response to IST. Additionally, it is approved for adults and children with chronic immune thrombocytopaenia (ITP) — for those patients who are refractory to other treatments and those with chronic hepatitis C virus (HCV) infection.
“Promacta is a great example of our drive to develop innovative treatments in serious disease areas where few treatment options exist,” said Dr Samit Hirawat, head, NovartisOncology Global Drug Development. “Thanks to the many individuals and organizations who have helped us to advance the development of this promising medicine. We will continue our work with the FDA to make Promacta available for this potential new indication as quickly as possible.”
The priority review designation that has been granted by the FDA has been based on the company’s recent analysis of research sponsored by the Intramural Research Program of the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) and conducted under a Cooperative Research and Development Agreement (CRADA).
In this study, it was demonstrated that more than half of treatment-naïve SAA patients achieved complete response at six months when treated with Promacta concurrently with standard IST, which was an increase of 35% compared to those treated with the standard IST alone. Additionally, overall response rate was 85% at six months.