halofuginone

Halofuginone is a fully synthetic small molecule and a low molecular weight quinazolinone alkaloid derived as a halogenated analog of febrifugine. It acts as a potent inhibitor of collagen alpha1(I) and matrix metalloproteinase 2 (MMP-2) gene expression, suppressing extracellular matrix deposition and cell proliferation. Its mechanism includes inhibition of Smad3 phosphorylation downstream of the TGFβ signaling pathway, which blocks fibroblast-to-myofibroblast transition and fibrosis. Halofuginone also inhibits prolyl-tRNA synthetase activity, affecting Th17 cell differentiation and modulating inflammation via the amino acid starvation response. The drug has demonstrated antitumor effects by inhibiting tumor stromal support, vascularization, invasiveness, and cell proliferation. Clinically developed for scleroderma (with orphan drug designation), Duchenne muscular dystrophy (as HT-100), coccidiosis in veterinary medicine (as Halocur), fibrosis-related diseases, autoimmune diseases involving Th17 cells, malaria (preclinical/experimental), cancer/fibrosis models in animals; it is rapidly absorbed orally with a half-life ranging from 23.8 to 72.1 hours[1][2][3][5][6].

Brand names

Halocur

Other names

halofuginone hydrobromidequinazolinone alkaloidsynthetic halogenated derivative of febrifugine