FDA grants accelerate approval for treatment of Chagas disease in pediatric patients

The U.S. Food and Drug Administration (FDA) recently granted accelerated approval to benzidazole for the treatment of Chagas disease in children aged 2 to 12 years old.

While the drug is one of the few treatments approved for use in adults, benzidazole now becomes the only approved treatment for the disease in children in the United States.

According to the FDA, safety and efficacy for benzidazole was established through two placebo-controlled clinical trials in children aged 6 to 12 years old, the first of which saw approximately 60 percent of trial participants treated with the drug have an antibody test change from positive to negative compared to approximately 14 percent in those who received a placebo.

In the second trial, approximately 55 percent of children treated with benzidazole had an antibody test change from positive to negative compared to five percent who received a placebo. A subsequent supplementary test of the treatment’s safety and pharmacokinetics revealed that the drug could be used in children as young as two years of age.

The accelerated approval designation signifies that the drug treats a serious medical condition where there is an unmet medical need. The designation is only granted after well-controlled clinical trials establish that the drug is likely to predict a benefit to patients.

Benzidazole was also granted priority review and orphan drug designation by the FDA, meaning the treatment addresses a rare disease and there were no drugs previously available to treat Chagas in children in the United States.

“The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases,” Edward Cox, director of the FDA’s Office of Antimicrobial Products, said.

Chagas is a tropical parasitic disease most commonly spread through insects known as tritominae. Typical symptoms of infection include fever, swollen lymph nodes, headaches and, more seriously, an enlargement of the ventricles of the heart, enlarged esophagus, and an enlarged colon.

According to a study published in the Lancet, approximately 8,000 patients worldwide succumb to the disease’s lethal effects each year.