New Help for Kids with Rare Blood Disorder

Promacta (eltrombopag) approved to treat low blood platelet count in kids with chronic immune thrombocytopenic purpura

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

(RxWiki News) Children with a rare blood disorder may soon get new help in the form of a pill.

The US Food and Drug Administration (FDA) Monday approved Promacta (eltrombopag) to treat low blood platelet count in children with chronic immune thrombocytopenic purpura (ITP), a rare blood disorder.

The agency approved the Novartis drug for kids older than 1 who have not seen results from other ITP drugs or spleen-removal surgery. Promacta was first approved in 2008 to treat adults with ITP.

“Today’s approval of Promacta emphasizes the FDA’s commitment to fully developing treatments in areas of pediatric hematology and oncology,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “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.”

In ITP, patients do not have enough of a type of cell that helps the blood clot (called platelets). A lack of platelets can lead to dangerous bleeding.

According to the FDA, Promacta counteracts low platelet counts by stimulating increased production. The once-daily tablet or powder appeared safe and effective in two studies of 159 ITP patients total. Among study patients taking Promacta, 62 percent saw an increase in platelets. In a group of ITP patients receiving a fake pill (placebo), only 32 percent saw an increase.

Patients in these studies reported side effects like respiratory infections, stomach pain, diarrhea and rash, among others.


Review Date: 
August 24, 2015
Last Updated:
August 31, 2015