The Food and Drug Administration (FDA) has approved Promacta (eltrombopag; Novartis), in combination with standard immunosuppressive therapy (IST) for the first-line treatment of adult and pediatric patients ≥2 years old with severe aplastic anemia (SAA).

The approval was based on data from a single-arm, open-label sequential cohort study in which patients received Promacta in combination with horse antithymocyte globulin (h-ATG) and cyclosporine; efficacy was established on the basis of complete hematological response at 6 months, defined as hematological parameters meeting all 3 of the following values on 2 consecutive serial blood count measurements at least 1 week apart: absolute neutrophil count (ANC) >1000/mcL, platelet count >100 x 109/L and hemoglobin >10g/dL, or reticulocyte count >60,000/mcL.

Results showed that 44% (95% CI 33, 55) of patients achieved complete response at 6 months while the overall response rate was 79% (95% CI 69, 87); the median duration of response was 24.3 months. The most common adverse reactions reported included abnormal liver function tests, rash, and skin discoloration.

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“Patients with SAA sometimes do not respond to the current treatment standard of IST,” said Phillip Scheinberg, MD, Head, Division of Hematology, Hospital A Beneficência Portuguesa de São Paulo in Brazil. “With this approval, physicians now have an option to add Promacta to the standard IST in a regimen that has demonstrated significant overall and complete response rates upfront in SAA and reduce the numbers of those who are unresponsive to initial therapy.”

Promacta, a thrombopoietin receptor agonist, is also indicated for: the treatment of patients with SAA who have had an insufficient response to IST; the treatment of thrombocytopenia in adult and pediatric patients ≥1 year with chronic immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy; and the treatment of thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy.

In addition to the new approval, Promacta has also been granted Breakthrough Therapy designation as a counter measure for hematopoietic sub-syndrome of acute radiation syndrome (H-ARS).

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