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.
“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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