Applied Therapeutics Provides Update Following Meeting with FDA

Core Viewpoint - Applied Therapeutics, Inc. has completed a meeting with the U.S. FDA regarding the potential new drug application for govorestat to treat Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) in Q3 2025 [1][2] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing transformative treatments for rare diseases [1][6] - The lead drug candidate, govorestat, is an Aldose Reductase Inhibitor aimed at treating multiple rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG [4][6] Disease Information - CMT-SORD is a rare hereditary neuropathy affecting approximately 3,300 patients in the U.S. and 4,000 patients in the EU, characterized by significant disability and loss of sensory function due to sorbitol accumulation [3] - The disease results from a deficiency of the enzyme sorbitol dehydrogenase, leading to high levels of sorbitol in blood and tissues [3] Regulatory Status - Govorestat has received Orphan Medicinal Product Designation from the EMA for both Galactosemia and CMT-SORD, and Orphan Drug Designation from the FDA for the same conditions [5] - The company is awaiting official meeting minutes from the FDA to determine the next steps for the NDA submission strategy, including potential eligibility for the accelerated approval pathway [2][5]