New Confirmatory Study Suggests Progress in Applied Therapeutics–FDA Alignment for Rare Disease Drug
Applied Therapeutics recently posted a new confirmatory study for its rare disease drug govorestat (an aldose reductase inhibitor) on ClinicalTrials.gov, suggesting possible renewed alignment with the FDA on the drug's development pathway for treating Charcot-Marie-Tooth sorbitol dehydrogenase deficiency (CMT-SORD)6.
The company has not yet officially confirmed whether this new study constitutes full alignment with the FDA, but market analysts suggest the move indicates a step forward in regulatory discussions6.
Earlier in September 2025, Applied Therapeutics announced that they met with the FDA to discuss the path for govorestat after emerging speculation about new clinical trial activity1.
No regulatory decision or agreement on accelerated approval has been finalized; Applied Therapeutics is awaiting official minutes from their recent FDA meeting before finalizing its regulatory submission plan1.
Govorestat previously missed its primary endpoint in a phase 2/3 trial, failing to show significant improvement over placebo in a 10-meter walk-run test, though this metric has since been removed from standard CMT assessments1.
In July 2025, Applied Therapeutics launched a free urine sorbitol assay program to support CMT-SORD diagnosis and management, indicating ongoing investment in this disease area3.
The FDA recently introduced new Rare Disease Evidence Principles (RDEP), creating a more flexible review pathway for ultra-rare conditions, in which single-arm trials and other supportive evidence may suffice for approval, potentially relevant to govorestat's pathway24.
Past regulatory hurdles included a December 2024 FDA rejection of govorestat for pediatric use, partially related to data and dosing management issues; the company has since experienced leadership changes and continues to push for approval in CMT-SORD1.
Sources:
1. https://www.fiercebiotech.com/biotech/after-surprise-trial-listing-applied-therapeutics-says-it-still-doesnt-have-any-updates-its
2. https://www.biopharmadive.com/news/fda-rare-disease-evidence-principles-drug-reviews/759237/
3. https://cmtrf.org/applied-therapeutics-to-meet-with-fda-on-new-drug-application-for-the-treatment-of-cmt-sord/
4. https://www.fiercebiotech.com/biotech/fda-proposal-would-allow-single-arm-trials-confirmatory-evidence-ultra-rare-diseases
6. https://www.biospace.com/drug-development/new-confirmatory-study-hints-at-alignment-between-applied-therapeutics-fda-for-rare-disease-drug