Late last night, the U.S. Food and Drug Administration (FDA) made waves when benznidazole, the country’s first sanctioned treatment for Chagas disease, was approved for use in children ages 2 to 12 years old.
Benznidazole, developed by Chemo Research, S.L., had previously been granted priority review and orphan product designation and it was approved using the Accelerated Approval pathway.
Chagas disease, a parasitic infection caused by Trypanosoma cruzi
, is carried by triatomine insects, also known as “kissing bugs.” It can be transmitted via touching feces of certain insects, blood transfusions, or from a mother to her child during pregnancy. Symptoms, after years of infection, can include potentially fatal heart disorders, and a negative effect on swallowing and digestion.
"The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases," said Edward Cox, M.D., director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research via a press release
Benznidazole was evaluated in 2 placebo-controlled clinical trials in pediatric patients ages 6 to 12 years old. Safety and efficacy were established after the first trial, when approximately 60% of children saw an antibody test change from positive to negative, compared to 14% with placebo. In the second trial, approximately 55% of children given benznidazole saw the same change in comparison to 5% of children given placebo.
Information on dosing recommendations for children from 2 to 12 years old was obtained via an additional study assessing the safety and pharmacokinetics of the drug.
Common adverse reactions of benznidazole in patients included stomach pain, rash, weight loss, headache, nausea, vomiting, irregular white blood cell count, hives, itchiness, and loss of appetite. Severe skin reactions, nervous system effects and bone marrow depression are also associated with the drug, and per findings in animal studies, benznidazole can cause fetal harm when administered to a pregnant woman.
With the approval, Chemo Research, S.L. was also awarded a Tropical Disease Priority Review Voucher, which can either be used for future drug applications or sold to other big pharma companies for several millions of dollars.
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