Drug intelligence / Profile preview

nifurtimox

Development stage
Approved
Lead developer
Bayer
Modality
Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Oral
01

Overview

Nifurtimox is an antiprotozoal small molecule drug primarily used to treat Chagas disease (American trypanosomiasis), an infection caused by the parasite *Trypanosoma cruzi*, especially in children and adolescents weighing at least 2.5 kg. It is also used as part of combination therapy for African trypanosomiasis (sleeping sickness). Nifurtimox belongs to the nitrofuran class and acts as a prodrug; its mechanism involves activation by parasite-specific type I nitroreductase enzymes, leading to the formation of cytotoxic metabolites that damage the parasite through multiple pathways, including inhibition of dehydrogenase activity and generation of reactive intermediates. The drug crosses the blood-brain barrier and placenta, but long-term use can cause significant gastrointestinal and neurological side effects[1][3][4][5][6].

Brand names
Lampit
02

Targets

GAPDH (Glyceraldehyde-3-phosphate dehydrogenase)Cyp (Cyclophilin family)NTR (Bacterial nitroreductase)

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