fosmetpantotenate

Fosmetpantotenate is a novel, investigational small molecule prodrug designed as a replacement therapy for pantothenate kinase-associated neurodegeneration (PKAN), a rare genetic disorder caused by mutations in the PANK2 gene. These mutations disrupt the biosynthesis of coenzyme A (CoA) by impairing the phosphorylation of vitamin B5 (pantothenate) to phosphopantothenic acid (PPA). Fosmetpantotenate is engineered to cross cell membranes and the blood-brain barrier, where it is converted into PPA, thereby bypassing the defective enzymatic step and restoring CoA levels. This restoration supports critical cellular functions such as energy metabolism, membrane integrity, and protein acetylation. Despite promising preclinical data and early clinical safety findings, fosmetpantotenate failed to demonstrate efficacy in Phase 3 trials for PKAN[1][5][6][7][8][9].

Other names

fosmetpantotenate