Fortunately, another drug has been approved by the US Food and Drug Administration (FDA) for children age one and over, a significant development for the cure of this rare blood disorder.
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Promacta can be used in the US in children aged one year and older when they have not achieved an appropriate response using other ITP medicines or surgery to remove the spleen.
Idiopathic thrombocytopenic purpura (ITP) is a disease that can cause easy bruising, bleeding and low levels of platelets in the blood making it hard for the blood to clot. Without enough platelets, bleeding can occur under the skin, in mucous membranes (such as in the mouth) or in other parts of the body.
“Today’s approval of Promacta emphasizes the FDA’s commitment to fully developing treatments in areas of pediatric hematology and oncology”, Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
Movartis said that the updated label will also include a new oral suspension formulation of Promacta that is designed for younger children who may not be able to swallow tablets.
The drug has not been evaluated among children less than a year old, the FDA said. Promacta, made by pharmacy giant Novartis, has been approved for the use of adults in 2008 and this June, it has been extended for children’s use from age six onwards.
ITP, which is characterized by a low platelet count, affects as many as five in 100,000 children each year. In a group of ITP patients receiving a fake pill (placebo), only 32 percent saw an increase.
In the second trial – composed of 92 patients – those who received eltrombopag daily for 13 weeks experienced a 41% increase in platelet counts for at least 6 out of 8 weeks between weeks 5 and 12, compared with 3% of patients who received placebo.
The trials showed that treatment with Promacta increased and sustained platelet counts among pediatric patients with chronic ITP with an insufficient response to prior chronic ITP therapies, and some patients taking concomitant ITP medications were able to reduce or discontinue their use of these medications, primarily corticosteroids. It holds orphan drug status.
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Eltrombopag originally developed by Swiss drug maker Novartis AG (ADR) (NYSE:NVS) is the base compound for Promacta®.
