Applied Therapeutics Tanks As FDA Path For Lead Rare-Disease Drug Turns Uncertain

Applied Therapeutics, Inc. (NASDAQ:APLT) stock is trading lower on Thursday, with a session volume of 20.07 million, compared to its average volume of 4.88 million, according to Benzinga Pro.

The company provided an update on regulatory strategy for Govorestat for Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) following receipt of Type C meeting minutes.

CMT-SORD is a genetic defect that prevents the body from metabolizing sorbitol, leading to its toxic buildup in cells and the resulting damage to peripheral nerves.

The company received official meeting minutes from the Type C meeting with the U.S. Food and Drug Administration (FDA), held in the third quarter of 2025, regarding Govorestat for CMT-SORD.

Key open issues included conclusive evidence for the pathophysiology of CMT-SORD, use of sorbitol levels as a surrogate endpoint, selection of a primary endpoint for a potential Phase 3 trial, and carcinogenicity testing.

Based on the official meeting minutes and post-meeting written correspondence, the company intends to submit a request for an additional Type C meeting with the FDA to further discuss a potential Phase 3 trial design.

The company has not determined the specific path forward for a potential regulatory approval submission, including whether a future FDA submission would be made under the accelerated approval pathway.

Cash and cash equivalents totaled $11.9 million as of September 30, 2025.

Price Action: APLT stock is down 58.8% at $0.37 at the last check on Thursday.

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