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AT-007
AT-007 is a small molecule that inhibits aldose reductase, the first enzyme in the polyol pathway, to reduce sorbitol accumulation and prevent diabetic complications.
AT-007 is a small molecule that inhibits aldose reductase, the first enzyme in the polyol pathway, to reduce sorbitol accumulation and prevent diabetic complications. Used for Diabetic peripheral neuropathy, Galactokinase deficiency.
At a glance
| Generic name | AT-007 |
|---|---|
| Also known as | aldose reductase inhibitor, Govorestat |
| Sponsor | Applied Therapeutics, Inc. |
| Drug class | Aldose reductase inhibitor |
| Target | Aldose reductase |
| Modality | Small molecule |
| Therapeutic area | Endocrinology / Diabetes |
| Phase | Phase 3 |
Mechanism of action
AT-007 blocks aldose reductase, which catalyzes the conversion of glucose to sorbitol in the polyol pathway. By inhibiting this enzyme, the drug reduces intracellular sorbitol accumulation in tissues affected by diabetes, thereby mitigating osmotic stress and oxidative damage that contribute to diabetic complications such as peripheral neuropathy and other microvascular disease.
Approved indications
- Diabetic peripheral neuropathy
- Galactokinase deficiency
Common side effects
- Headache
- Nausea
- Diarrhea
Key clinical trials
- Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia (PHASE2, PHASE3)
- Confirmatory Study of Govorestat in CMT-SORD (PHASE3)
- Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency (PHASE2, PHASE3)
- Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia (PHASE1, PHASE2)
- AT-007 in Adult Subjects With Classic Galactosemia (CG) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AT-007 CI brief — competitive landscape report
- AT-007 updates RSS · CI watch RSS
- Applied Therapeutics, Inc. portfolio CI