FDA approves use of Promacta in young kids suffering from rare blood disorder
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. For treatment, most medical experts recommend surgery if the situation is critical and medications for more moderate to serious cases. 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.
Daily RX writes that last Monday, the FDA has announced that it is extending the use of Promacta for children with ITP for the ages one year and above to help curb low blood platelet count. This is especially vital for children who did not respond well with other drugs. 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.
Dr. Richard Pazdur, the director of the Office of Hematology and Oncology products at the FDA's Center for Drug Evaluation and Research, said that with the approval of the drug for children's use is to highlight the FDA's commitment to look for possible development and treatment in the field of pediatric hematology and oncology. Dr. Padzur further explained that the approval could be the answer for the children who can't seem to get well with current available treatment in the market.
Furthermore, Street Insider writes that the approval also included an oral suspension formula in case younger children are unable to swallow the tablets.
The extension of the approval has been brought about by the results of two double-blind trials which include a Phase 3 clinical trial in almost 160 patients. The research suggests that Promacta is able to increase and maintain platelet counts among kids with chronic ITP even after being treated before with ITP therapies. The result also shows kids taking corticosteroids are able to reduce taking this kind of treatment and have gradually improved which led to discontinued use.
eNews Park Forest adds that although there has been noted adverse effects like upper respiratory tract infection, diarrhea, rash, increased liver enzymes, abdominal pain and nose or throat infection, it is considered by the FDA for approval because it is an orphan drug designation. This means it treats a rare disease and as it provides financial incentives, there is more means to further studies concerning this condition.
It is noted that there is still no studies done to check the safety and efficacy of Promacta in kids younger than one year old.