Medication Monitor



Generic Name (Trade Name—Company)
Notes
August 30, 2017

Benznidazole

(‚ÄéRochagan, Radanil—Chemo Research, S. L.)
FDA approves first U.S. treatment for Chagas disease

FDA granted accelerated approval to benznidazole for use in children aged 2 years to 12 years with Chagas disease. It is the first treatment approved in the United States for treatment of this disease.

Chagas disease, or American trypanosomiasis, is a parasitic infection caused by Trypanosoma cruzi. It can be transmitted through different routes, including contact with the feces of a certain insect, blood transfusions, or from a mother to her child during pregnancy. After years of infection, the disease can cause serious heart illness, and it also can affect swallowing and digestion. While Chagas disease primarily affects people living in rural parts of Latin America, recent estimates are that there may be approximately 300,000 persons in the United States with the disease.

Safety and efficacy of benznidazole were established in two placebo-controlled clinical trials in pediatric patients aged 6 years to 12 years old. In the first trial, approximately 60% of children treated with benznidazole had an antibody test change from positive to negative compared with approximately 14% of children who received a placebo.

Results in the second trial were similar: Approximately 55% of children treated with benznidazole had an antibody test change from positive to negative compared with 5% who received a placebo.

An additional study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2 years to 12 years provided information for dosing recommendations down to 2 years of age. 

The most common adverse reactions were stomach pain, rash, decreased weight, headache, nausea, vomiting, abnormal white blood cell count, urticaria, pruritus, and decreased appetite.

Benznidazole is associated with serious risks, including serious skin reactions, nervous system effects, and bone marrow depression. Based on findings from animal studies, benznidazole could cause fetal harm when administered to a pregnant woman.