An evaluation of nifurtimox for Chagas disease in children

LASCANO, FERNANDA; ALTCHEH, J

Expert Opinion on Orphan Drugs

Taylor & Francis

Lugar: Londres; Año: 2021

Introduction: Chagas Disease (CD) is a zoonosis disease caused by Trypanosoma Cruzi (T.cruzi) and currently affects 6 million people worldwide. Described 100 years ago, twodrugs are available for treatment, Nifurtimox (NFX) and Benznidazole (BZN), developedover 50 years ago. Though BZN has been more commonly used for CD, new developmentefforts on NFX has been undertaken with its recently released pediatric formulation and anongoing study continuation with long term follow-up. Areas covered: In this narrativereview we searched electronic databases (Eg PUBMED, COCHRANE) and criticallyanalyzed clinical and pre-clinical studies about NFX for CD treatment from a pediatricperspective, and focusing on pharmacodynamics, pharmacokinetics, metabolism, safety,tolerance, and clinical efficacy of this drug.Expert opinion: In order to achieve CD elimination goals, early diagnosis and opportunetreatment are fundamental. In this context the approval by FDA of NFX for children 0 to 18years of age has made this drug the only one available in 0 to 2 years and over 12 agegroups in USA. This approval might also change preconceptions about NFX safety andtolerance. Considering CD has only two pharmacological options, this is a significant steptowards a new era for the treatment of this disease in the pediatric population.Key words: chagas disease treatment, children, chagas disease drugs, pediatric clinicalpharmacology, nifurtimox, Trypanosoma cruzi.