Charcot-Marie-Tooth Association’s Strategy To Accelerate Research (CMTA-STAR) Alliance Partner Applied Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted the company a meeting this fall to discuss its plan to submit its investigational drug govorestat (AT-007) for approval to treat CMT-SORD. This type of request is called a New Drug Application, or NDA, and it is required for the FDA to consider approving a treatment.
Govorestat is the company’s investigational drug it is studying to treat CMT-SORD, also known as Sorbitol Dehydrogenase (SORD) deficiency. This potential treatment for CMT-SORD targets the buildup of a sugar called sorbitol, which becomes toxic to peripheral nerves. In people with CMT-SORD, mutations in the SORD gene cause sorbitol levels to rise to damaging levels, leading to muscle weakness, sensory loss, and other symptoms associated with this form of Charcot-Marie-Tooth disease.
Sponsored Sorbitol Testing Now Available
Applied Therapeutics also announced that it has launched a sponsored urine sorbitol testing program to help healthcare providers identify patients with suspected CMT-SORD. This urine test, called a polyol urinalysis and available from Mayo Clinic (test code SORD), checks specific sugar levels, including sorbitol. In CMT-SORD, sorbitol levels are very high in urine and can confirm a suspected CMT-SORD diagnosis.
The company reports that this sponsored testing is available at no cost to patients and caregivers, removing potential barriers to diagnosis, care management, and future treatment.
A Shared Commitment
Applied Therapeutics recently presented full 12-month results from its INSPIRE Phase 2/3 trial at the 2025 annual meeting of the Peripheral Nerve Society, along with 18- and 24-month data showing slowed disease progression on MRI. Govorestat treatment also improved key clinical and patient-reported outcomes.
“This FDA meeting is an exciting next step toward making a treatment for CMT-SORD a reality,” said CMTA CEO Sue Bruhn, PhD. “We are proud to support our CMTA-STAR Alliance Partner Applied Therapeutics in advancing govorestat, and we remain committed to accelerating progress that brings effective treatments to everyone in the CMT community.”
Looking Ahead
If the NDA submission process moves forward, it will represent a major milestone for the CMT-SORD community and for everyone working toward approved treatments for CMT. CMTA will continue to share updates on the progress of govorestat CMT-SORD research and other projects through our CMTA-STAR program.
Read Applied Therapeutics’ full release here.
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Published on: August 14, 2025