Applied Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for govorestat (AT-007) for the treatment of Classic Galactosemia by three months. The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024. The FDA notified Applied Therapeutics that it required additional time to review supplemental analyses of previously submitted data that had been provided by Applied in response to the FDA's routine information requests and determined that the additional information constitutes a Major Amendment to the NDA.

In February 2024, the Company announced that the FDA accepted and granted Priority Review to the NDA. Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval. The NDA filing of govorestat is supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile.

The submission package included clinical outcomes data from the Phase 3 registrational 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. If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics' first commercial product. Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and SORD Deficiency.

Govorestat has also received Orphan Drug Designation from the U.S. Food & Drug Administration (FDA) for the treatment of Galactosingmia, PMM2-CDG, and SORD Deficiency; Pediatric Rare Disease designation for Galactosemia and PMM2-CDG; and Fast Track designation for Galactosemia.