The drug is the first treatment approved in the US for Chagas disease.
The US Food and Drug Administration (FDA) granted an accelerated approval to Chemo Research S.L.'s benznidazole for use against Chagas disease in children aged 2 to 12 years. It is the first treatment approved Chagas disease treatment in the United States.
The approval was granted based on 2 placebo-controlled clinical trials in which the results showed that the safety and efficacy of benznidazole in established pediatric patients 6 to 12 years old, according to a press release from the FDA. Chagas disease, or American trypanosomiasis, is a parasitic infection caused by Trypanosoma cruzi.
With this approval, Chemo Research, S. L. has been awarded a Tropical Disease Priority Review Voucher in accordance with a provision included in the Food and Drug Administration Amendments Act of 2007 that aims to encourage the development of new drugs and biological products for the prevention and treatment of certain tropical diseases.
“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 statement.
In the first trial, about 60% of children treated with benznidazole had an antibody test change from positive to negative compared with only 14% of children who received a placebo. The second trial's results were similar findings that approximately 55% of children treated with benznidazole experienced an antibody test change from positive to negative compared with 5% in the placebo arm.
An additional study of the safety and pharmacokinetics of benznidazole in pediatric patients aged 2 to 12 provided information for dosing recommendations down to 2 years of age.
There were some adverse reactions for patients receiving benznidazole, the most common being stomach pain, rash, decreased weight, headache, nausea, vomiting, abnormal white blood cell count, urticaria (hives), pruritus (itching), and decreased appetite. Benznidazole is also associated with some serious risks, including serious skin reactions, nervous system effects, and bone marrow depression.
"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 Chagas disease," according to the FDA.