Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for govorestat, for the treatment of CMT-SORD. If the New Drug Application process advances, this could be a pivotal step toward making the therapy available to patients outside of clinical trials.
CMT-SORD is a rare axonal subtype of CMT caused by mutations in the SORD gene. These mutations impair the function of an enzyme responsible for breaking down a sugar metabolite called sorbitol, leading to its accumulation. Elevated levels of sorbitol is toxic to the nerve cells and leads to neuromuscular deficits and other symptoms related to the disease.
Govorestat is a small molecule drug that aims to reduce the toxic levels of sorbitol by inhibiting an enzyme responsible for converting sugar into sorbitol.
Additionally, in July 2025, Applied Therapeutics launched a free Urine Sorbitol Assay — a lab test that measures sorbitol levels in urine to help identify patients with CMT-SORD. This program is intended to support healthcare providers in identifying suspected cases of CMT-SORD through urine-based sorbitol measurement using a clinically validated and compliant testing workflow. The Urine Sorbitol Assay is available to healthcare providers at no cost for patients and caregivers, removing potential barriers to diagnosis, care management and treatment.
“CMTRF is encouraged by Applied Therapeutics’ continued progress toward a treatment for CMT-SORD,” said Laura M. MacNeill, CMTRF’s CEO. “The upcoming FDA meeting and launch of the free sorbitol assay are important milestones that bring hope to our patient community, helping speed diagnosis and move us closer to a targeted therapy that could slow disease progression and improve quality of life for those living with this rare form of CMT.”
In May 2025, Applied Therapeutics presented full 12-month clinical results and topline 18-and 24-month patient level analyses from the INSPIRE Phase 2/3 trial of govorestat at the PNS 2025 Annual Meeting. To learn more about the INSPIRE Phase 2/3 trial findings, visit https://cmtrf.org/applied-therapeutics-presents-findings-from-phase-2-3-clinical-trial-of-govorestat-in-cmt-sord/
To learn more about the potential NDA, visit https://appliedtherapeutics.gcs-web.com/news-releases/news-release-details/applied-therapeutics-reports-second-quarter-2025-financial
To learn more about the Global CMT Research Convention, visit https://cmtconvention.cmtrf.org/