FDA grants accelerated approval to benznidazole for Chagas disease
The FDA granted accelerated approval to benznidazole for the treatment of Chagas disease among children aged 2 to 12 years old in the United States.
With this approval, benznidazole (Chemo Research, S. L.) is the first U.S.-approved treatment for Chagas disease, a parasitic infection caused by Trypanosoma cruzi, which can be transmitted through triatomine bugs — or “kissing bugs” — as well as blood transfusions and vertical transmission during pregnancy.
Once endemic only in Latin America, Chagas disease, and its associated disease vector, has continued to spread to Europe and the U.S., with more than 300,000 cases recorded in the U.S. Although many patients may remain asymptomatic — and therefore undiagnosed — Chagas cardiomyopathy occurs in 20% to 30% of patients, with the potential to progress to dilated cardiomyopathy and congestive heart failure.
“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.
The FDA based its approval on two placebo-controlled clinical trials that evaluated safety and efficacy among pediatric patients aged 6 to 12 years. In the initial trial, antibody tests for approximately 60% of patients treated with benznidazole changed from positive to negative, compared with 14% of patients who received placebo.
The secondary trial reported similar results, with approximately 55% of patients experiencing an antibody tests change from positive to negative, compared with 5% of patients who received placebo. A supplementary study of the safety and pharmacokinetics of benznidazole among pediatric patients provided data for dosing recommendations for children as young as 2 years of age.
The most common adverse events associated with the use of benznidazole included stomach pain, rash, decreased weight, headache, nausea, vomiting, abnormal white blood cell count, urticaria, pruritus and decreased appetite. Additionally, benznidazole has previously been associated with risks such as serious skin reactions, nervous system effects and bone marrow depression; based on data from animal studies, benznidazole may cause fetal harm if administered to pregnant women.