Benznidazole granted accelerated approval for use in children aged 2 to 12 years with Chagas disease.
The FDA on Tuesday granted accelerated approval to benznidazole for pediatric patients aged 2 to 12 years with Chagas disease. This is the first drug approved in the United States to treat the condition, according to a press release.
Chagas disease is a parasitic infection caused by Trypanosoma cruzi and can be transmitted through insect feces, blood, or from mother-to-child. After years of untreated infection, Chagas disease can result in problems with swallowing, digestion, and the heart.
Although Chagas disease typically affects individuals in Latin America, it is projected that more than 300,000 individuals have the condition in the United States, according to the FDA.
“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” said Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research.
The safety and efficacy of benznidazole was evaluated in 2 clinical trials of pediatric patients.
In the first study, 60% of children treated with benznidazole had an antibody change from positive to negative, while only 14% of children treated with placebo achieved the results, according to the study.
The results from the second study were similar. The investigators reported that 55% of children treated with benznidazole had the antibody test change compared with only 5% of placebo-treated patients.
Another pharmacokinetics study determined the dosing information for pediatric patients, according to the release.
Common adverse events include stomach pain, rash, weight loss, headache, nausea, vomiting, abnormal white blood cell count, urticaria, pruritus, and decreased appetite.
The FDA warns that benznidazole may result in serious risks, such as skin reactions, nervous system effects, and bone marrow depression. The drug may also cause fetal harm, as evident by animal studies, according to the release.
Benznidazole received accelerated approval, due to lack of treatment for Chagas disease; however, additional studies are needed to further confirm the clinical benefits of the drug.
The drug was also granted priority review and orphan drug designation since Chagas disease is a rare condition and there are no approved therapies.
The manufacturer, Chemo Research, SL, will receive a Tropical Disease Priority Review Voucher as a way to encourage the creation of novel treatments for the prevention and treatment of tropical diseases, according to the FDA.