he FDA has expanded the approval of eltrombopag (Promacta) in combination with standard immunosuppressive therapy (IST) to include newly diagnosed adult and pediatric patients 2 years and older with severe aplastic anemia.
The FDA has expanded the approval of eltrombopag (Promacta) in combination with standard immunosuppressive therapy (IST) to include newly diagnosed adult and pediatric patients 2 years and older with severe aplastic anemia (SAA), according to Novartis, the manufacturer of eltrombopag.1 The agency also granted the agent a breakthrough therapy designation as a counter measure for hematopoietic sub-syndrome of acute radiation syndrome (H-ARS).
The approval is based on a Novartis analysis sponsored by the National Heart, Lung, and Blood Institute (NHLBI) Division of Intramural Research Program and conducted under a Cooperative Research and Development Agreement. Findings showed that treatment with eltrombopag given concurrently with standard IST led to an overall response rate (ORR) of 79% (95% CI, 69%-87%) and a 44% complete response (CR) rate in patients with definitive IST-naïve SAA, both at 6 months. This compares with a 27% CR rate historically observed with standard IST alone.
"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, and previously with the NHLBI. "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."
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