Applied Therapeutics (NASDAQ:APLT) saw its stock dive 55% in premarket trading Monday after the company disclosed uncertainty around the regulatory pathway for govorestat, its therapy for Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD).
The clinical-stage biopharmaceutical firm recently met with the U.S. Food and Drug Administration (FDA) to discuss a potential new drug application for govorestat. However, the company highlighted that a “path forward regarding a potential submission strategy for regulatory approval, including under the accelerated approval pathway, has not been determined.”
Applied Therapeutics said it is still awaiting the official FDA meeting minutes to clarify the next steps for the drug, which targets CMT-SORD, a rare hereditary neuropathy affecting roughly 3,300 patients in the U.S. and 4,000 in Europe.
The company had previously aimed for a potential NDA submission in the third quarter of 2025. Despite the recent setback, Applied Therapeutics reaffirmed its dedication to meeting the needs of patients living with CMT-SORD.
Govorestat, a central nervous system-penetrant Aldose Reductase Inhibitor, has received multiple regulatory designations, including Orphan Medicinal Product Designation from the European Medicines Agency for both Galactosemia and CMT-SORD, as well as Orphan Drug Designation from the FDA for several rare conditions.
Applied Therapeutics stock price
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