Article · Wikipedia archive · Last revised Jun 15, 2026

Govorestat

Govorestat (AT-007) is an aldose reductase inhibitor and experimental drug to treat galactosemia and sorbitol dehydrogenase deficiency.

Last revised
Jun 15, 2026
Read time
≈ 1 min
Length
314 w
Citations
6
Source
Wikipedia ↗
Govorestat
Clinical data
Other namesAT-007
ATC code
Identifiers
  • 2-[4-Oxo-3-[[5-(trifluoromethyl)-1,3-benzothiazol-2-yl]methyl]thieno[3,4-d]pyridazin-1-yl]acetic acid
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
ChEMBL
Chemical and physical data
FormulaC17H10F3N3O3S2
Molar mass425.40 g·mol−1
3D model (JSmol)
  • C1=CC2=C(C=C1C(F)(F)F)N=C(S2)CN3C(=O)C4=CSC=C4C(=N3)CC(=O)O
  • InChI=1S/C17H10F3N3O3S2/c18-17(19,20)8-1-2-13-12(3-8)21-14(28-13)5-23-16(26)10-7-27-6-9(10)11(22-23)4-15(24)25/h1-3,6-7H,4-5H2,(H,24,25)
  • Key:ORQGHAJIWGGFJK-UHFFFAOYSA-N

Govorestat (AT-007) is an aldose reductase inhibitor and experimental drug to treat galactosemia1 and sorbitol dehydrogenase deficiency.2

After a report circulating on the internet accused the developer Applied Therapeutics of cutting corners in its studies of the drug, the FDA put a hold on it in 2020. Applied Therapeutics said that the report was a fraudulent attempt to manipulate its stock price.3

In February 2024, the FDA granted priority review states to an application from Applied Therapeutics to use govorestat to treat galactosemia, a metabolic disease.4 The FDA then rejected the company’s application in November 2024, and issued a warning letter to then-President Shoshana Shendelman informing her of “objectionable conditions” observed during an FDA inspection earlier that year.5

Society and culture

In December 2024, Advanz Pharma Limited withdrew its application for a marketing authorization of Nugalviq for the treatment of classic galactosaemia, a condition where the body cannot break down a sugar called galactose.6

References

References

  1. Bailey, Evan; Wang, Stella; Saltonstall, Laura; Perfetti, Riccardo; Shendelman, Shoshana (March 2022). "OP005: AT-007 significantly reduces toxic galactitol in ACTION-galactosemia kids - the 1st therapeutic interventional clinical trial in children with classic galactosemia". Genetics in Medicine. 24 (3): S336. doi:10.1016/j.gim.2022.01.556. S2CID 247603696.
  2. "CTG Labs - NCBI". clinicaltrials.gov. 22 September 2023. Retrieved 5 November 2023.
  3. "Bad to worse for Applied Therapeutics as FDA slaps partial clinical hold on rare drug". Retrieved 4 November 2023.
  4. "Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia". Applied Therapeutics. 28 February 2024.
  5. Research, Center for Drug Evaluation and (3 December 2024). "Applied Therapeutics, Inc. - 696833 - 12/03/2024". FDA. Retrieved 8 July 2025.
  6. "Nugalviq EPAR". European Medicines Agency (EMA). 10 December 2024. Retrieved 16 February 2025.