The FDA has accepted its 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 anemia (SAA).
This morning, Novartis announced that the US Food and Drug Administration has accepted its 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 anemia (SAA).
Eltrombopag is an oral thrombopoietin receptor agonist (TPO-RA).
SAA is a rare and lethal blood disorder. It is characterized by the failure in a patient's bone marrow to produce enough red blood cells, white blood cells, and platelets. Mortality rates are typically between 80% and 90% in SAA patients with death occurring between 1 and 2 years.
"Promacta is a great example of our drive to develop innovative treatments in serious disease areas where few treatment options exist," commented Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development in a recent statement.
Novartis' analysis of a research study sponsored by the Intramural Research Program of the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) and led under a Cooperative Research and Development Agreement (CRADA) served as the basis for eltrombopag’s Priority Review for first-line SAA.
In the study, when treated with Promacta in combination with standard IST, over half (52%) of treatment-naïve SAA patients obtained a complete response at 6 months. This treatment combination exhibited an increase of 35% compared to those treated with the standard IST alone. At 6 moths, 85% experienced an overall response rate.
"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."
Previously, eltrombopag was approved for SAA patients who have had an insufficient response to IST in the refractory setting as well as for adults and children with chronic immune thrombocytopenia (ITP) for patients who are refractory to other treatments and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus (HCV) infection.
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