The first treatment for Chagas disease in the United States has been approved by the US Food and Drug Administration (FDA). Benznidazole was granted accelerated approval for use in children between the ages of two and 12 years old. The drug also received priority review and orphan product designation due to the scarcity of Chagas disease in the US and the fact that no medication is currently available.
Accelerated approval allows the FDA to approve a medicine for serious conditions in circumstances where there is an unmet medical need and where trials have demonstrated the "an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients". The FDA stated that while approval has been granted, further study is required to verify and describe the benefits of benznidazole. Benznidazole is manufactured by Chemo Research and was awarded a Tropical Disease Priority Disease Review Voucher.
Commenting on the latest approval, Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research, said: "The FDA is committed to making available safe and effective therapeutic options to treat tropical diseases."
Benznidazole's safety and efficacy were established in two placebo-controlled clinical trials. A further study examined the safety and pharmacokinetics of the drug in patients aged 2-12 and provided information regarding the recommendations for dosages. The most common adverse reactions to the medication were: stomach pain, rash, weight loss, headaches, nausea, vomiting, abnormal white blood cell count, hives, itching, and a decreased appetite.
Chagas disease, also known as American trypanosomiasis, is a parasitic infection with a number of routes of transmission, including blood transfusion, certain insect feces, or from mother to child during pregnancy. It can lead to health complications such as heart issues and problems swallowing and digesting.
It manifests in two phases: the first is an acute phase lasting around two months, in which symptoms are generally mild or absent. During the second, chronic phase, patients may suffer cardiac disorders or digestive issues as the parasites are hidden in the heart and digestive muscles. In addition to treatment with benznidazole, the drug nifurtimox, which is available under the trade name Lampit, may also be used.
The disease is one of five neglected parasitic infections targeted by the Centers for Disease Control and Prevention (CDC) as a condition for which public health action is needed. Estimates from the CDC suggest up to 300,000 people in the US have the Trypanosoma cruzi infection. According to the World Health Organization, between six and seven million people across the world, the majority of whom are in Latin America, are infected with the parasite.
