Chagas disease: First time approval of a treatment in the US
Last Updated on August 31, 2017 by Joseph Gut – thasso
August 31, 2017 – For the very first time, the American Food & Drug Administration (FDA) granted approval for a treatment of Chagas disease in the the United States. Chagas disease, or American trypanosomiasis, is a parasitic infection caused by Trypanosoma cruzi and can be transmitted through different routes, including contact with the feces of a certain insect, blood transfusions, or from a mother to her child during pregnancy.
After years of infection, the disease can cause serious heart illness, and it also can affect swallowing and digestion. While Chagas disease primarily affects people living in rural parts of Latin America, recent estimates are that there may be approximately 300,000 persons in the United States with Chagas disease. Chagas disease is considered a “tropical disease” which normally would not be found in the US spontaneously. However, in the age of worldwide large scale immigration- and emmigration- movements of people for whatever reasons, movements of people for economical reasons, and large scale deplacement of populations as a consequence of warfare, in every day medicine at any one place, it becomes a true challenge to provide the best possible therapeutic option for a given condition.
With the approval of Benznidazole, the FDA has committed to making available a safe and effective therapeutic option to treat this tropical disease in the US. The FDA approved Benznidazole for use in children ages 2 to 12 years old with Chagas disease. Interestingly, outside of the US, Benznidazole is on the World Health Organization’s List of Essential Medicines, the most effective and safe medicines needed in a global health system, and thereby is addresssing the need of Chagas disease patients worldwide.
The safety and efficacy of Benznidazole were established in two placebo-controlled clinical trials in pediatric patients 6 to 12 years old. In the first trial, approximately 60 percent of children treated with Benznidazole had an antibody test change from positive to negative compared with approximately 14 percent of children who received a placebo. Results in the second trial were similar: Approximately 55 percent of children treated with Benznidazole had an antibody test change from positive to negative compared with 5 percent who received a placebo. An additional study of the safety and pharmacokinetics (how the body absorbs, distributes and clears the drug) 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. Based on findings from animal studies, Benznidazole could cause fetal harm when administered to a pregnant woman. According to the FDA drug label for Benznidazole, it comes with contraindications such as a history of hypersensitivity reaction to benznidazole or other nitroimidazole derivatives, a disulfiram usage within the last two weeks, and alcoholic beverage consumption during and for at least three days after therapy.