AT-007

Applied Therapeutics, Inc. · Phase 3 active Small molecule

AT-007 is a Aldose reductase inhibitor Small molecule drug developed by Applied Therapeutics, Inc.. It is currently in Phase 3 development for Diabetic peripheral neuropathy, Galactokinase deficiency. Also known as: aldose reductase inhibitor, Govorestat.

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 an experimental drug that has been studied for its potential to treat Classic Galactosemia, Hereditary Neuropathy Caused by SORD Deficiency, and Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency. It is an aldose reductase inhibitor, a type of drug that works by inhibiting the aldose reductase enzyme.

Likelihood of approval

58.3% vs 58.3% industry baseline

If approved by FDA: likely 2028–2030

Steps remaining: NDA/BLA submission

Confidence: High

Why this estimate

• Baseline phase 3 → approval rate +58.3pp
  Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2028–2030	—
EMA	EU	2029–2031	+0.7 yr
MHRA	GB	2029–2031	+0.7 yr
Health Canada	CA	2029–2032	+0.9 yr
TGA	AU	2029–2032	+1.2 yr
PMDA	JP	2029–2032	+1.5 yr
NMPA	CN	2030–2033	+2.3 yr
MFDS	KR	2029–2032	+1.4 yr
CDSCO	IN	2029–2033	+1.8 yr
ANVISA	BR	2030–2033	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

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

Frequently asked questions about AT-007

What is AT-007?

AT-007 is a Aldose reductase inhibitor drug developed by Applied Therapeutics, Inc., indicated for Diabetic peripheral neuropathy, Galactokinase deficiency.

How does AT-007 work?

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.

What is AT-007 used for?

AT-007 is indicated for Diabetic peripheral neuropathy, Galactokinase deficiency.

Who makes AT-007?

AT-007 is developed by Applied Therapeutics, Inc. (see full Applied Therapeutics, Inc. pipeline at /company/applied-therapeutics-inc).

Is AT-007 also known as anything else?

AT-007 is also known as aldose reductase inhibitor, Govorestat.

What drug class is AT-007 in?

AT-007 belongs to the Aldose reductase inhibitor class. See all Aldose reductase inhibitor drugs at /class/aldose-reductase-inhibitor.

What development phase is AT-007 in?

AT-007 is in Phase 3.

What are the side effects of AT-007?

Common side effects of AT-007 include Headache, Nausea, Diarrhea.

What does AT-007 target?

AT-007 targets Aldose reductase and is a Aldose reductase inhibitor.

Related

• Drug class: All Aldose reductase inhibitor drugs
• Target: All drugs targeting Aldose reductase
• Manufacturer: Applied Therapeutics, Inc. — full pipeline
• Therapeutic area: All drugs in Endocrinology / Diabetes
• Indication: Drugs for Diabetic peripheral neuropathy
• Indication: Drugs for Galactokinase deficiency
• Also known as: aldose reductase inhibitor, Govorestat