Applied Therapeutics Announces MAA Validation and NDA Submission of Govorestat (AT-007) for Treatment of Classic Galactosemia

NDA submitted to FDA and MAA validated by EMA in December 2023

Submissions are based on positive results from Phase 3 registrational ACTION-Galactosemia Kids study demonstrating consistent long-term clinical outcomes benefit across a range of functional measures and favorable safety profile

NEW YORK, Jan. 03, 2024 (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 it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was submitted in December 2023. In addition, the Company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fourth quarter of 2023, which was subsequently validated and accepted for review in December 2023.

“The submissions of both the NDA and MAA for govorestat are 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,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We look forward to working closely with both regulatory agencies throughout the review process and hope to bring the first treatment to patients with Galactosemia soon.”

“There are currently no treatments available for Galactosemia, a serious progressive disease affecting over 3,000 patients in the United States,” said Brittany Cudzilo, Vice President of the Galactosemia Foundation. “Govorestat offers hope to many patients and families affected by Galactosemia. I saw the positive impact of govorestat first-hand in my daughter, Ansell, who participated in the ACTION-Galactosemia Kids pediatric study. As a community, we will do everything possible to ensure that the regulatory agencies understand the importance of approving this treatment for our loved ones with Galactosemia.”

The NDA and MAA submission packages include clinical outcomes data from the Phase 3 registrational ACTION-Galactosemia Kids study in children age 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. The MAA has been validated and will move to review by the EMA’s Committee for Medicinal Products for Human Use (CHMP).