Applied Therapeutics (APLT, Financials) announced that the U.S. Food and Drug Administration issued a Complete Response Letter regarding its New Drug Application for govorestat, a treatment for Classic Galactosemia; the stock closed at $2.03, down 76.3%.
Citing flaws in the clinical application, the FDA said it cannot accept the NDA as it is now presented.
The business said it is going over FDA comments and will call a conference to go over criteria for possible resubmission or appeal of the ruling.
Founder and CEO Shoshana Shendelman expressed disappointment, stating, "We are disappointed by the FDA's decision today. Our strong commitment to the Galactosemia community is rooted in our belief that govorestat has the potential to change the lives of patients with Galactosemia."
During clinical studies, Govorestat, a central nervous system penetrant aldose reductase inhibitor, has rapidly and consistently lowered galactitol levels, producing favorable results for young patients. The Phase 3 ACTION-Galactosemia Kids trial found advantages in activities of daily living, conduct, cognition, and motor abilities among other areas. Over three years, more studies including adults and children produced safety and supported effectiveness evidence.
With an NDA filing expected for early 2025, Govorestat is also being developed to treat uncommon neuromuscular condition Sorbitol Dehydrogenase.
The hereditary condition known as galactosemia prevents galactose from being metabolized, which causes neurological problems. Clinically based Applied Therapeutics specializes on rare illness therapies. Govorestat for many metabolic disorders makes up its pipeline.
