Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia

PDUFA target action date of August 28, 2024

MAA validated and accepted for review by EMA in December 2023

NEW YORK, Feb. 28, 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 that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) 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.

“The FDA’s acceptance of the NDA for govorestat for the treatment of Galactosemia represents a critical milestone for Applied Therapeutics and more importantly, for patients with Galactosemia and their families. The Agency’s decision to grant Priority Review for this NDA underscores the urgent unmet medical need as there are currently no treatment options for this devastating disease,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We want to thank the patients, families, collaborators and physicians involved in reaching this achievement. We look forward to continuing to work with the FDA throughout the review process, as we hope to bring govorestat to patients as quickly as possible.”

“We are excited to see that the FDA has accepted the govorestat NDA for priority review,” said Nicole Casale, President of the Galactosemia Foundation. “The Galactosemia community is in urgent need of a safe and effective treatment, and the potential approval of govorestat provides hope for many families. Acceptance of the govorestat NDA brings us one step closer to availability of a first-ever treatment.”

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 3 registrational ACTION-Galactosemia Kids study in children aged 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data.