FDA extends use of Promacta in young children with rare blood disorder

The US Food and Drug Administration late on August 24 approved Promacta (eltrombopag) to treat low blood platelet count in pediatric patients (aged one year and older) with a rare blood disorder - chronic immune thrombocytopenic purpura (ITP).

Promacta, from Swiss pharma giant Novartis (NOVN: VX), can be used in these children when they have not achieved an appropriate response using other ITP medicines or surgery to remove the spleen, the FDA stated and noted that drug has not been evaluated among children less than a year old.

“Today’s approval of Promacta emphasizes the FDA’s commitment to fully developing treatments in areas of pediatric hematology and oncology,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This new use in ages one and up builds on a recent approval for ages six years and up, and fills an unmet need for young children whose disease has progressed after use of other available treatments,” he noted.