Govorestat
An investigational treatment for Charcot-Marie-Tooth Disease.
The same compound appears under different names depending on the context. Here is how to identify Govorestat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Govorestat
- Development code
- AT-007
- Drug class
- Aldose reductase inhibitor (oral small molecule)
- Manufacturer
- Cycle Pharmaceuticals (Applied Therapeutics)
- How it's taken
- Taken by mouth as an oral capsule, once daily.
An investigational oral aldose reductase inhibitor in development for CMT-SORD, a recessive form of Charcot-Marie-Tooth disease caused by mutations in the SORD gene. Its Phase 2/3 INSPIRE trial finished in June 2026, and results have not been posted. The same molecule was studied in classic galactosemia, where the FDA issued a Complete Response Letter in November 2024.
Where Govorestat fits
Investigational, not yet approved. If approved, would be the first FDA-approved disease-modifying therapy for any CMT subtype. Only relevant for patients with biallelic SORD mutations confirmed on genetic testing, so confirming subtype with a broad gene panel is the first step.
How Govorestat works
CMT-SORD is caused by changes (mutations) in both copies of a gene called SORD. The SORD gene normally produces an enzyme that helps clear sorbitol, a sugar-alcohol that the body makes from glucose. When SORD is broken, sorbitol cannot be cleared properly. It builds up in peripheral nerve cells and damages them, causing the slowly progressive weakness and sensory loss patients experience.
Govorestat works one step further upstream. It blocks aldose reductase, the enzyme that converts glucose into sorbitol in the first place. By stopping the build-up at the source, govorestat lowers the toxic sorbitol levels that drive the nerve damage. Treatment is taken as a once-daily pill, which makes it different from the AAV gene therapies being tested for other CMT subtypes (those require injections into the spinal fluid).
If the INSPIRE Phase 2/3 trial confirms benefit, govorestat would be the first drug specifically approved for CMT-SORD, and the first FDA-approved disease-modifying therapy for any subtype of Charcot-Marie-Tooth disease.
Mechanism: Aldose reductase inhibitor that blocks the enzyme converting glucose into sorbitol. In CMT-SORD, where the SORD gene is broken, sorbitol builds up to nerve-damaging levels. Govorestat lowers sorbitol back toward normal so the nerves can recover or stop deteriorating.
Side effects and safety
Because govorestat is still in clinical trials for CMT-SORD, the full safety picture is being established. The same molecule has been studied in late-stage trials for classic galactosemia, where the most common side effects reported were headache, diarrhea, and abdominal pain. The CMT-SORD trial is collecting safety data on a different patient population, so the profile may differ. The trial team will share complete safety information when the trial reads out.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Govorestat
Taken by mouth as an oral capsule, once daily. Specific dosing for adults and pediatric patients with CMT-SORD is being established through the INSPIRE Phase 2/3 trial.
Clinical trial results
[INSPIRE (NCT05397665)](https://clinicaltrials.gov/study/NCT05397665) was the pivotal Phase 2/3 trial of govorestat in patients with confirmed CMT-SORD (biallelic SORD mutations). The trial measured changes in nerve function and patient-reported function over time and compared govorestat to placebo. ClinicalTrials.gov lists it as completed in June 2026, with no results posted yet. A separate confirmatory Phase 3 study was withdrawn before enrolling anyone.
Development history
Govorestat (then called AT-007) was originally developed by Applied Therapeutics for classic galactosemia. After researchers identified that mutations in SORD cause a treatable form of CMT through the same sorbitol-buildup mechanism, Applied Therapeutics expanded the development program to CMT-SORD. The company hit FDA setbacks in galactosemia (a Complete Response Letter on November 27, 2024) but the CMT-SORD program is on a separate regulatory path with its own data. Cycle Pharmaceuticals completed its acquisition of Applied Therapeutics in February 2026.
Explore Charcot-Marie-Tooth Disease trials
Other Charcot-Marie-Tooth Disease treatments
Common questions about Govorestat
▸What is Govorestat?
An investigational oral aldose reductase inhibitor in development for CMT-SORD, a recessive form of Charcot-Marie-Tooth disease caused by mutations in the SORD gene. Its Phase 2/3 INSPIRE trial finished in June 2026, and results have not been posted. The same molecule was studied in classic galactosemia, where the FDA issued a Complete Response Letter in November 2024.
▸How does Govorestat work?
CMT-SORD is caused by changes (mutations) in both copies of a gene called SORD. The SORD gene normally produces an enzyme that helps clear sorbitol, a sugar-alcohol that the body makes from glucose. When SORD is broken, sorbitol cannot be cleared properly. It builds up in peripheral nerve cells and damages them, causing the slowly progressive weakness and sensory loss patients experience.
Govorestat works one step further upstream. It blocks aldose reductase, the enzyme that converts glucose into sorbitol in the first place. By stopping the build-up at the source, govorestat lowers the toxic sorbitol levels that drive the nerve damage. Treatment is taken as a once-daily pill, which makes it different from the AAV gene therapies being tested for other CMT subtypes (those require injections into the spinal fluid).
If the INSPIRE Phase 2/3 trial confirms benefit, govorestat would be the first drug specifically approved for CMT-SORD, and the first FDA-approved disease-modifying therapy for any subtype of Charcot-Marie-Tooth disease.
▸What are the side effects of Govorestat?
Because govorestat is still in clinical trials for CMT-SORD, the full safety picture is being established. The same molecule has been studied in late-stage trials for classic galactosemia, where the most common side effects reported were headache, diarrhea, and abdominal pain. The CMT-SORD trial is collecting safety data on a different patient population, so the profile may differ. The trial team will share complete safety information when the trial reads out.
▸How is Govorestat taken?
Taken by mouth as an oral capsule, once daily. Specific dosing for adults and pediatric patients with CMT-SORD is being established through the INSPIRE Phase 2/3 trial.
▸Is Govorestat FDA approved?
Govorestat is currently in phase 3 clinical trials for Charcot-Marie-Tooth Disease. It has not yet received FDA approval.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Applied Therapeutics. CMT-SORD Program. https://www.appliedtherapeutics.com/cmt-sord/
- U.S. National Library of Medicine — ClinicalTrials.gov. INSPIRE: A Study of AT-007 in Subjects With Sorbitol Dehydrogenase Deficiency (SORD-d). https://clinicaltrials.gov/study/NCT05397665
- Hereditary Neuropathy Foundation. Charcot-Marie-Tooth Disease — Clinical Trials. https://www.hnf-cure.org/our-impact/clinical-trials/
- Applied Therapeutics · 2024-11-27. Applied Therapeutics Receives Complete Response Letter from U.S. FDA Regarding New Drug Application for Govorestat for Classic Galactosemia. https://www.globenewswire.com/news-release/2024/11/27/2988438/0/en/Applied-Therapeutics-Receives-Complete-Response-Letter-from-U-S-FDA-Regarding-New-Drug-Application-for-Govorestat-for-Classic-Galactosemia.html
- Applied Therapeutics / SEC · 2026-02-03. Cycle Pharmaceuticals Completes Acquisition of Applied Therapeutics. https://www.sec.gov/Archives/edgar/data/1697532/000119312526034514/d216453dex99a5k.htm