FDA Grants Accelerated Approval for First US Chagas Disease Treatment
Chagas disease can result in cardiovascular morbidity and mortality if left untreated. Photo Credit: CDC/ Dr Mae Melvin.
The US Food and Drug Administration (FDA) has granted accelerated approval for benznidazole, the first treatment of Chagas disease in the United States, according to a press release.1
Chagas disease, commonly found in rural parts of Latin America, is a parasitic infection caused by Trypanosoma cruzi transmitted via insect feces, blood transfusions, and in utero. An estimated 300,000 people in the United States have Chagas disease, which can cause serious heart illness and affect swallowing and digestion.
Benznidazole, manufactured by Chemo Research SL, was evaluated in 2 placebo-controlled clinical trials in children aged 6 to 12 years. In the first trial, almost 60% of participants treated with benznidazole had antibody tests change from positive to negative compared with 14% of participants who received placebo. In the second trial, approximately 55% of participants treated with benznidazole had antibody tests change from positive to negative. Another study provided additional information regarding safety, pharmacokinetics, and dosing recommendations for treatment in pediatric participants, lowering the minimum age for treatment to 2 years.2
The FDA approved benznidazole using the Accelerated Approval pathway and granted the treatment priority review and orphan product designation since Chagas disease is a rare disease and there was no approved treatment in the United States until recently.
Serious risks have been reported in patients taking benznidazole, including serious skin reactions, nervous system adverse effects, and bone marrow depression. Animal studies with benznidazole prompt concern that the treatment could cause fetal harm when administered in pregnant women. Therefore, further study is needed to verify benznidazole's clinical benefit.
- FDA approves first US treatment for Chagas disease [press release]. Silver Spring, Maryland: US Food and Drug Administration. Published August 29, 2017. Accessed August 30, 2017.
- Francisco AF, Jayawardhana S, Lewis MD, Taylor MC, Kelly JM. Biological factors that impinge on Chagas disease drug development [August 23, 2017]. Parasitology. 2017:1-10. doi: 10.1017/S0031182017001469