Biology:Govorestat
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
Legal status
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
- ↑ 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.
- ↑ CTG Labs - NCBI. 22 September 2023. https://clinicaltrials.gov/study/NCT05397665. Retrieved 5 November 2023.
- ↑ "Bad to worse for Applied Therapeutics as FDA slaps partial clinical hold on rare drug". https://www.fiercebiotech.com/biotech/bad-to-worse-for-applied-therapeutics-as-fda-slaps-partial-clinical-hold-rare-drug.
- ↑ "Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia". Feb 28, 2024. https://appliedtherapeutics.gcs-web.com/news-releases/news-release-details/applied-therapeutics-announces-fda-acceptance-and-priority.
- ↑ Research, Center for Drug Evaluation and (2024-12-03). "Applied Therapeutics, Inc. - 696833 - 12/03/2024" (in en). https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/applied-therapeutics-inc-696833-12032024.
- ↑ "Nugalviq EPAR". 10 December 2024. https://www.ema.europa.eu/en/medicines/human/EPAR/nugalviq.
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