New Indication for Promacta for Severe Aplastic Anemia

First-Line Treatment for People With Life-Threatening Blood Disorder

The FDA has expanded the use of Promacta (eltrombopag, Novartis) to include first-line treatment for patients from the age of two years who have severe aplastic anemia (SAA). Eltrombopag will be used in combination with standard immunosuppressive therapy (IST) and is the first new treatment in decades for newly-diagnosed SAA patients in the U.S.

Eltrombopag, an oral thrombopoietin receptor agonist, is designed to boost low platelet counts via once-daily tablets and is already approved for SAA in patients who had an insufficient response to IST. The drug is also approved for adults and children with chronic immune thrombocytopenia that is resistant to other treatments, and for treating thrombocytopenia in patients with chronic hepatitis C virus(HCV) infection.

People with SAA can suffer from such debilitating symptoms and complications as fatigue, difficulty breathing, recurring infections, and abnormal bruising or bleeding, which can severely limit their daily activities.

The FDA based the new indication on results of NHLBI-sponsored research that included immunosuppressive therapy-naive patients with severe aplastic anemia. The most common adverse reactions reported among at least 5% of the study population included abnormal liver function tests, rash and skin discoloration, including hyperpigmentation.

In addition, the FDA also granted Promacta breakthrough therapy designation as a countermeasure for hematopoietic sub-syndrome (bone marrow syndrome) of acute radiation syndrome. Research has indicated that the drug could decrease the risk of hemorrhage in patients with radiation sickness.

Sources:, November 20, 2018;, November 20, 2018.