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Classic Galactosemia

1
Pipeline Programs
1
Companies
4
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Applied Therapeutics
1 program
1
AT-007Phase 34 trials
Active Trials
NCT05397665Active Not Recruiting56Est. Oct 2026
NCT05418829Unknown7Est. Dec 2022
NCT04902781Completed47Est. May 2025
+1 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Applied TherapeuticsAT-007
Applied TherapeuticsAT-007
Applied TherapeuticsAT-007
Applied TherapeuticsAT-007

Clinical Trials (4)

Total enrollment: 224 patients across 4 trials

NCT05418829Applied TherapeuticsAT-007

AT-007 in Adult Subjects With Classic Galactosemia (CG)

Start: Oct 2021Est. completion: Dec 20227 patients
Phase 3Unknown
NCT05397665Applied TherapeuticsAT-007

Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency

Start: Jan 2022Est. completion: Oct 202656 patients
Phase 2/3Active Not Recruiting
NCT04902781Applied TherapeuticsAT-007

Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia

Start: Mar 2021Est. completion: May 202547 patients
Phase 2/3Completed
NCT04117711Applied TherapeuticsAT-007

Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

Start: Jun 2019Est. completion: Dec 2021114 patients
Phase 1/2Completed

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
1 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.