NIH study in NEJM shows Novartis drug eltrombopag as first-line therapy with standard treatment improves responses in severe aplastic anemia

Novartis International AG / NIH study in NEJM shows Novartis drug eltrombopag as first-line therapy with standard treatment improves responses in severe aplastic anemia . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

• NIH study found 58% of patients with treatment-naïve severe aplastic anemia (SAA) achieved a complete response when eltrombopag was given at the initiation of and concurrently with standard immunosuppressive therapy[1]
   

The historical complete response rate was 10% for untreated patients with SAA on immunosuppressive therapy alone[1] 
 

Findings were published in The New England Journal of Medicine (NEJM) and will be submitted to regulatory bodies for treatment-naïve SAA

Basel, April 19, 2017 - Novartis today announced the publication of a study conducted by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) demonstrating that 58% of patients with treatment-naïve severe aplastic anemia (SAA) achieved complete response at six months when treated with eltrombopag at the initiation of and concurrently with standard immunosuppressive treatment[1]. The study evaluated three sequential treatment groups, or cohorts. Cohort 3 added eltrombopag at the initiation of immunosuppressive therapy and showed a higher complete response rate than cohorts 1 and 2, where eltrombopag was initiated on day 14. The data is published in the latest issue of The New England Journal of Medicine.

SAA is a rare and serious blood disorder in which a patient's bone marrow fails to make enough red blood cells, white blood cells and platelets[2]. As a result, people living with SAA may experience debilitating symptoms and complications, such as fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can limit their daily activities[2]. The current standard of care includes immunosuppressive therapy (IST) or hematopoietic stem cell transplantation. However, one-quarter to one-third of patients will not respond to IST and 30-40% of responders will relapse, causing symptoms to return[3].