Applied Therapeutics Announces Successful Pre-NDA Meeting with the FDA and Confirms Plans to Submit NDA for Govorestat (AT-007) for Treatment of Classic Galactosemia to FDA in Q4 2023

NEW YORK, Sept. 06, 2023 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced successful completion of a recent pre-New Drug Application (“NDA”) meeting with the Food and Drug Administration (FDA) regarding its govorestat (AT-007) Galactosemia program. Based on discussions with the FDA, the Company believes they are aligned with the FDA and plans to submit an NDA for govorestat (AT-007) for the treatment of Galactosemia in the fourth quarter of this year.

“In the pre-NDA meeting, the FDA expressed their support for a potential NDA based on the govorestat data generated to date in Galactosemia, and provided constructive recommendations for successful NDA acceptance and review,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “If approved, govorestat will fill an important unmet need for the Galactosemia community. We will work to prepare our regulatory package as expeditiously as possible and expect to submit the NDA to the FDA in the fourth quarter of this year.”

“We thank the FDA for their productive feedback and for their partnership in this process,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “We believe that the clinical efficacy demonstrated to date, combined with galactitol biomarker data and a favorable safety profile, provides us with a solid filing package to the FDA. We are excited to advance towards a regulatory submission for the first potential treatment for Galactosemia.”

About Galactosemia
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 3,000 patients with Galactosemia in the US and 80 new births per year, and approximately 4,000 patients with Galactosemia in the EU and 120 new births per year.