FDA acceptance and priority review of NDA for govorestat for the treatment of classic galactosemia

The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024. The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application. Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.

Govorestat is an investigational, novel Aldose Reductase Inhibitor (ARI) being developed for the treatment of several rare diseases. It is a potent and selective compound, which crosses the blood brain barrier into the Central Nervous System (CNS). 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 III registrational ACTION-Galactosemia Kids study in children aged 2-17 with Galactosemia, the Phase 1/II 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. The FDA grants Priority Review to applications for potential medicines that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition. Govorestat for the treatment of Galactosemia has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation, Pediatric Rare Disease Designation and Fast Track Designation from the FDA. The Company has also submitted a Marketing Authorization Application (MAA) for govorestat for the treatment of Classic Galactosemia to the EMA, which was validated in December 2023 and is under review by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The Company expects a decision by the EMA in the fourth quarter of 2024.