Govorestat

Govorestat
Clinical data
Other namesAT-007
ATC code
Identifiers
IUPAC name
  • 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)
SMILES
  • C1=CC2=C(C=C1C(F)(F)F)N=C(S2)CN3C(=O)C4=CSC=C4C(=N3)CC(=O)O
InChI
  • 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 galactosemia[1] 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

  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.
This article is issued from WikiProjectMed. The text is available under Creative Commons Attribution-ShareAlike License unless otherwise noted. Additional terms may apply for the media files.