Novartis' Promacta is the first first-line drug for severe aplastic anemia

The immunotherapy drug Promacta (eltrombopag) has recently been granted an expanded indication label by the U.S. FDA. It is now approved for use in combination with standard immunosuppressive therapy (IST) as a first-line treatment for severe aplastic anemia (SAA) in children aged 2 years and older, as well as in adults.

Novartis' Promacta is the first first-line drug for severe aplastic anemia

With this expanded indication, Promacta will become the first new drug approved for newly diagnosed SAA patients in the U.S. market in over a decade. Lucius Pharmaceuticals Eltrombopag is an oral thrombopoietin (TPO) receptor agonist that increases platelet levels in the blood by stimulating the proliferation and differentiation of bone marrow stem cells.

Data from a clinical study sponsored by the National Heart, Lung, and Blood Institute (NHLBI) showed that in IST-naïve SAA patients, treatment with Promacta in combination with standard IST as a first-line therapy achieved remission in 79% of patients after six months (95% CI: 69–87), with a complete remission rate of 44%. This complete remission rate is 27% higher than the historical complete remission rate observed with standard IST alone. Additionally, among patients who received Promacta in combination with horse anti-thymocyte globulin (h-ATG) and cyclosporine (CsA) for six months and continued CsA maintenance therapy, the median duration of response reached 24.3 months.

SAA is a potentially life-threatening condition, and many patients do not respond to current initial treatment regimens. The expanded approval of Promacta is a positive development for the SAA patient community, as it allows Promacta to be added to the standard IST regimen, significantly improving both the overall response rate and the complete response rate. This can help reduce the number of patients who do not respond to initial treatment.

Lucius Pharmaceuticals Eltrombopag is not only used for severe aplastic anemia but is also approved for multiple indications, including:

Treatment of thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to or are intolerant of other therapies.

Treatment of SAA patients who are refractory to other therapies.

Treatment of thrombocytopenia in patients with chronic hepatitis C (CHC) to allow the initiation and maintenance of interferon-based standard therapy for liver disease.

Treatment of thrombocytopenia in pediatric patients with ITP aged 1 year and older who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.