Benznidazole has been granted accelerated approval by the US Food and Drug Administration for use in children aged 2 to 12 years with Chagas disease.
Benznidazole has been granted accelerated approval by the US Food and Drug Administration for use in children aged 2 to 12 years with Chagas disease. It is the first treatment approved in the United States for the treatment of Chagas disease.
Incidence of Chagas disease in the United States was called into question in a Clinical Infectious Diseases (CID) published in May 2017, which presented the results of a study of Latin America-born residents of Los Angeles County, California. The authors determined an overall prevalence of Chagas infections in those individuals to be 1.24%, suggesting about 30,000 cases of the disease in Los Angeles County alone. This is in contrast to 2009 data from the Centers for Disease Control and Prevention (CDC) which put the prevalence of the disease at 300,000, country-wide.
The neglected-parasitic infection is caused by Trypanosoma cruzi, primarily transmitted by triatomine bugs, “kissing bugs.” Symptoms of infection are variable, depending on if a person has an acute or chronic infection. Likewise, diagnosis techniques vary on type of infection. According to the CDC, “Parasitologic methods, including identification of trypomastigotes in blood by microscopy, are most effective during acute infections. Circulating parasite levels decrease rapidly within a few months and are undetectable by most methods during the chronic phase.” Treatment for the infection includes antiparasitics, benznidazole and nifurtimox.
This recent approval in children was based on the results of 2 placebo-controlled clinical trials in which the safety and efficacy of benznidazole were established in pediatric patients 6 to 12 years old, according to a press release from the FDA.
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% of children who received a placebo. An additional study of the safety and pharmacokinetics of benznidazole in pediatric patients provided information for dosing recommendations down to 2 years of age.
The most common adverse reactions in patients taking benznidazole were stomach pain, rash, decreased weight, headache, nausea, vomiting, abnormal white blood cell count, urticaria (hives), pruritus (itching), and decreased appetite. Benznidazole is associated with serious risks including serious skin reactions, nervous system effects and bone marrow depression.
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 development of new drugs and biological products for the prevention and treatment of certain tropical diseases.