It is the first treatment approved in the United States for the treatment of Chagas disease.
Officials with the US FDA have granted accelerated approval to Chemo Research S.L.'s benznidazole 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.
The approval was based on the results of 2 placebo-controlled clinical trials in which the safety and efficacy of benznidazole were established 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% who received a placebo. An additional study of the safety and pharmacokinetics of benznidazole in pediatric patients 2 to 12 years of age 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.
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 development of new drugs and biological products for the prevention and treatment of certain tropical diseases.
According to reporting in Pharmacy Times' sister website, Contagion.com a study published on May 1, 2017 in the journal Clinical Infectious Diseases(CID) noted overall Chagas prevalence in the United States may be about 1.24%. This study suggested that there may be as many as 30,000 cases of Chagas in Los Angeles County alone.1
Meymandi SK, Forsyth CJ, Soverow J, et al. Prevalence of Chagas Disease in the Latin American—born Population of Los Angeles. Clin Infect Dis. 2017 May 1; 64(9): 1182—1188.