The Food and Drug Administration announced Aug. 29 the accelerated approval of benznidazole for use in children aged 2-12 years who have Chagas disease.
In two placebo-controlled clinical trials, researchers examined pediatric patients aged 6-12 with Chagas disease. In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative, compared with about 14% children who received a placebo. Similar findings were found in the second trial. In that one, about 55% of children treated with benznidazole had an antibody test change from positive to negative, compared with 5% who received a placebo. Also, a different study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2-12 provided information for dosing recommendations down to 2 years of age.
Chagas disease, an infection caused by the Trypanosoma cruzi parasite, primarily affects people living in rural parts of Latin America. However, recent estimates suggest that about 300,000 people in the United States may have the disease. After infection, people commonly experience either no or mild symptoms, according to the FDA. Some people infected with the parasite go on to develop major heart or gastrointestinal tract problems.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.
This is the first treatment approved in the United States for Chagas disease. According to the agency, additional study is needed to “verify and describe the anticipated clinical benefit of benznidazole.”