Inborn Errors of Metabolism
1
Pipeline Programs
3
Companies
3
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
1
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Human BioSciencesWV - Martinsburg
2 programs1
N-carbamylglutamatePhase 2/31 trial
Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry ProtocolN/A
Active Trials
CENTOGENEGermany - Rostock
1 programBiomarkers for Inborn Errors of MetabolismN/A1 trial
Active Trials
Genome & CompanyKorea - Suwon
1 programMaternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry ProtocolN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Human BioSciencesN-carbamylglutamate
CENTOGENEBiomarkers for Inborn Errors of Metabolism
Genome & CompanyMaternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
Clinical Trials (3)
Total enrollment: 516 patients across 3 trials
N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia
Start: Aug 2008Est. completion: Jun 202052 patients
Phase 2/3Unknown
Biomarkers for Inborn Errors of Metabolism
Start: Aug 2019Est. completion: Mar 2022462 patients
N/ACompleted
NCT02322177Genome & CompanyMaternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol
Start: Dec 2014Est. completion: Aug 20182 patients
N/ACompleted
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
3 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.