FDA Expedites Review of Promacta for Severe Aplastic Anemia

Eltrombopag (Promacta) was granted Priority Review designation, indicating a 6-month timeline for review.

Today Novartis announced that the US Food and Drug Administration (FDA) accepted the supplemental New Drug Application and granted Priority Review status to eltrombopag (Promacta) combined with standard immunosuppressive therapy for first-line treatment of severe aplastic anemia.

Severe aplastic anemia is a life-threatening condition where a patient’s bone marrow does not produce sufficient red blood cells, white blood cells, and platelets. These patients may experience fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can limit their daily activities.

Earlier this year, eltrombopag was granted Breakthrough Therapy designation by the FDA, highlighting the need for a treatment for this rare blood disorder.

"Promacta is a great example of our drive to develop innovative treatments in serious disease areas where few treatment options exist," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development.

The Priority Review designation for first-line severe aplastic anemia (SAA) is based on the 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).

The study showed that 52% of treatment-naïve SAA patients achieved complete response at 6 months when treated with eltrombopag concurrently with standard immunosuppressive therapy (IST), which was an increase of 35% compared to patients treated with standard IST alone. The overall response rate to eltrombopag with standard IST was 85% at 6 months.

Eltrombopag is an oral thrombopoietin receptor agonist that is already approved for SAA in the refractory setting for patients who have had an insufficient response to IST, for adults and children with chronic immune thrombocytopenia for patients who are refractory to other treatments, and for the treatment of thrombocytopenia in patients with chronic hepatitis C virus infection.

"Thanks to the many individuals and organizations who have helped us to advance the development of this promising medicine,” said Hirawat. “We will continue our work with the FDA to make Promacta available for this potential new indication as quickly as possible."