Applied Therapeutics announced that the U.S. FDA has declined to approve its New Drug Application for govorestat, a novel, central nervous system (CNS)-penetrant aldose reductase inhibitor (ARI) for the treatment of Classic Galactosemia.
According to the drugmaker, the complete response letter cited "deficiencies in the clinical application."
Applied shared what appeared to be a positive update back in September, revealing that the FDA had communicated that an Advisory Committee meeting would no longer be required and that the PDUFA date of November 28 was still on track.
Now, the company says it is reviewing the feedback from the FDA and plans to immediately request a meeting to discuss requirements for a potential resubmission of the NDA or appeal of the decision along with appropriate next steps.
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. In the phase 3 ACTION-Galactosemia Kids study in children with Galactosemia aged 2-17, treatment with govorestat demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremors. Govorestat also significantly reduced plasma galactitol levels in both adults and children with Galactosemia.
Govorestat is also being developed for the treatment of Sorbitol Dehydrogenase (SORD) Deficiency, a rare and progressive neuromuscular disease. The company expects to submit an NDA early in the first quarter of 2025.