NEW YORK - Applied Therapeutics, Inc. (NASDAQ:APLT), a biopharmaceutical company, announced the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for govorestat (AT-007), a treatment for Classic Galactosemia, with a Priority Review designation. The FDA has scheduled a Prescription Drug User Fee Act (PDUFA) target action date for August 28, 2024, and plans to convene an advisory committee meeting to discuss the application.
Govorestat, an investigational Aldose Reductase Inhibitor (ARI), has been granted Pediatric Rare Disease designation, potentially qualifying it for a Priority Review Voucher (PRV) upon approval. The acceptance of the NDA by the FDA marks a significant step for Applied Therapeutics and patients with Galactosemia, a rare genetic metabolic disorder that currently has no approved treatment options.
The submission for govorestat is supported by data from the Phase 3 ACTION-Galactosemia Kids study and the Phase 1/2 ACTION-Galactosemia study, indicating a reduction in galactitol levels and improvements in clinical outcomes for pediatric patients, coupled with a favorable safety profile.
If approved, govorestat would be the first medication indicated for Galactosemia treatment and Applied Therapeutics' inaugural commercial product. The company has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), which was validated in December 2023 and is under review, with a decision expected in the fourth quarter of 2024.
Galactosemia is characterized by the inability to metabolize galactose, leading to neurological complications, juvenile cataracts, and ovarian insufficiency in women. Approximately 3,000 patients in the U.S. and 4,000 in the EU live with this condition.
The FDA grants Priority Review to drug applications that could significantly improve treatment, prevention, or diagnosis of a serious condition. Govorestat has also received Orphan Medicinal Product Designation from the EMA and Orphan Drug Designation, Pediatric Rare Disease Designation, and Fast Track Designation from the FDA.
This news is based on a press release statement from Applied Therapeutics, Inc.
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