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# Review Extended on Govorestat NDA
- URL: https://www.fdaweb.com/review-extended-on-govorestat-nda/
- Published: 2024-03-29T12:00:00.000Z
- Updated: 2026-09-14T14:26:52.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156668

FDA has extended by three months its review of an Applied Therapeutics NDA for govorestat (AT-007) for treating classic galactosemia. The agency review extension gives it additional time to review supplemental analyses of previously submitted data that had been provided by Applied in response to FDA’s routine information requests, according to the company. Govorestat is described as a central nervous system penetrant aldose reductase inhibitor. The new user fee review action target date is 11/28.

Galactosemia is a rare genetic metabolic disease where patients are unable to metabolize the simple sugar galactose, according to the company. “When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women),” it says.

Applied Therapeutics says the submission is based on clinical outcomes data from the Phase 3 ACTION-Galactosemia Kids study in children aged 2-17 with galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with galactosemia, and preclinical data. It adds that treatment with govorestat “demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor. Govorestat also significantly reduced plasma galactitol levels in both adults and children with galactosemia.”