govorestat

Govorestat (AT-007) is a potent, orally active, central nervous system (CNS) penetrant small molecule aldose reductase inhibitor (ARI) being developed by Applied Therapeutics. It is primarily indicated for the treatment of Charcot-Marie-Tooth disease with sorbitol dehydrogenase deficiency (CMT-SORD), a rare and progressive hereditary neuropathy. In patients with CMT-SORD, mutations in the SORD gene result in the inability to metabolize sorbitol, leading to its toxic accumulation in peripheral nerves and motor neurons. Govorestat acts by inhibiting aldose reductase, the enzyme responsible for converting glucose into sorbitol, thereby significantly lowering sorbitol levels and potentially slowing or halting disease progression. The drug has demonstrated clinical efficacy in reducing blood sorbitol levels and improving functional outcomes in Phase 2/3 trials, and it has received Orphan Drug Designation from both the FDA and EMA.

Brand names

Nugalviq

Other names

AT-007AT007AT 007