FDA grants breakthrough therapy designation for severe aplastic anemia drug


The Food and Drug Administration has granted breakthrough therapy designation to eltrombopag (Promacta) for use in combination with standard immunosuppressive therapy as a first-line treatment for patients with severe aplastic anemia (SAA).

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The breakthrough therapy designation – which provides expedited review for drugs for serious or life-threatening conditions – is based on results from a phase 1-2 study conducted by researchers at the National Heart, Lung, and Blood Institute. In all, 52% of newly treated patients with SAA achieved a complete response at 6 months when treated with eltrombopag at the start of and concurrently with standard immunosuppressive treatment, according to an analysis of the data conducted by Novartis, which sponsors the drug.

Eltrombopag already is approved as a second-line therapy in patients with refractory SAA and is approved for adults and children with refractory chronic immune thrombocytopenia (ITP).

“Promacta is a promising medicine that, if approved for first-line use in severe aplastic anemia, may redefine the standard of care for patients with this rare and serious bone marrow condition,” Samit Hirawat, MD, head of Novartis Oncology Global Drug Development, said in a statement.

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