Govorestat Explained

Atc Prefix:None
Cas Number:2170729-29-8
Pubchem:132260161
Unii:6JLQ8K35KK
Drugbank:DB16707
Synonyms:AT-007
Chemspiderid:81409264
Chembl:4650327
Stdinchi: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)
Stdinchikey:ORQGHAJIWGGFJK-UHFFFAOYSA-N
Smiles:C1=CC2=C(C=C1C(F)(F)F)N=C(S2)CN3C(=O)C4=CSC=C4C(=N3)CC(=O)O
Iupac Name:2-[4-Oxo-3-[[5-(trifluoromethyl)-1,3-benzothiazol-2-yl]methyl]thieno[3,4-d]pyridazin-1-yl]acetic acid
C:17
H:10
F:3
N:3
O:3
S:2

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]

Notes and References

  1. Bailey . Evan . Wang . Stella . Saltonstall . Laura . Perfetti . Riccardo . Shendelman . Shoshana . 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 . March 2022 . 24 . 3 . S336 . 10.1016/j.gim.2022.01.556. 247603696 . free .
  2. Web site: CTG Labs - NCBI . clinicaltrials.gov . 5 November 2023.
  3. News: Bad to worse for Applied Therapeutics as FDA slaps partial clinical hold on rare drug . 4 November 2023.