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# Applied Therapeutics NDA Denied Approval
- URL: https://www.fdaweb.com/applied-therapeutics-nda-denied-approval/
- Published: 2024-12-02T12:00:00.000Z
- Updated: 2026-09-14T14:48:16.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158284

FDA has issued Applied Therapeutics a complete response letter on its NDA for govorestat, a central nervous system-penetrant aldose reductase inhibitor (ARI) for treating classic galactosemia. The letter cited “deficiencies in the clinical application,” the company says, adding that it is requesting a meeting with the agency to discuss requirements for a potential NDA resubmission or an appeal of the decision.

Galactosemia is described as a rare genetic metabolic disease that makes it difficult to metabolize the simple sugar galactose. When not metabolized properly, galactose is converted to a toxic metabolite, galactitol, which causes neurological complications, including deficiencies in cognition, behavior, activities of daily living, adaptive skills, fine and gross motor skills and speech, as well as tremor and seizures, the company says.

In clinical trials, govorestat has shown “rapid and sustained reductions in galactitol,” Applied Therapeutics says. “In the Phase 3 registrational ACTION-Galactosemia Kids study in children with galactosemia aged 2-17, treatment with govorestat demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor," it says. "Govorestat also significantly reduced plasma galactitol levels in both adults and children with galactosemia.”