FDA Approves Low Blood Platelet Drug for Children

The FDA has approved an sNDA for Novartis’ Promacta for the treatment of children six years and older with chronic immune thrombocytopenia who have had a poor response to corticosteroids, immunoglobulins or spleen removal.

The drug was originally approved in 2008 for use in adults with the same low blood platelet disorder. The once-daily oral thrombopoietin receptor agonist works by stimulating large cells found in bone marrow to increase platelet production.

The approval was based clinical trials that showed 63 percent of cITP patients taking Promcata had increased and sustained platelet counts. Further, 14 percent of patients who needed other cITP medications — primarily corticosteroids — were able to reduce or eliminate their use of those treatments, Novartis says.

Promcata (eltrombopag) was acquired by Novartis from GlaxoSmithKline in March. It was discovered as part of a collaboration between GSK and Ligand Pharmaceuticals and developed by GSK.
cITP affects roughly five in 100,000 children annually. — Kellen Owings