The Food and Drug Administration (FDA) has approved benznidazole (Chemo Research) for the treatment of Chagas disease in patients 2–12 years of age. This accelerated approval marks the first treatment for Chagas disease in the U.S.
Chagas disease, also known as American trypanosomiasis, is caused by the Trypanosoma cruzi parasite. It can be transmitted via fecal contact from a certain insect, blood transfusions, or from a mother to a child during pregnancy.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” said Edward Cox, M.D., director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research.
The safety and efficacy of benznidazole were established in two placebo-controlled trials in patients aged 6–12 years. In one trial, about 60% of children who received benznidazole had an antibody test change from positive to negative vs. 14% of children who received placebo. In the second trial, about 55% of children who received benznidazole had an antibody test change from positive to negative vs. 5% of children who received placebo.
A separate safety and pharmacokinetics study of benznidazole in patients aged 2–12 years provided the data for recommended dosing for those as young as 2 years old.
Stomach pain, rash, decreased weight, headache, nausea, vomiting, abnormal white blood cell count, urticaria, and pruritus were among the most commonly seen adverse reactions with benznidazole.
Previously, benznidazole was granted Priority Review and Orphan Product designations by the FDA.
For more information visit FDA.gov.