Molybdenum Cofactor Deficiency, Type A
2
Pipeline Programs
1
Companies
2
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
1
1
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
OriginORGN001
OriginORGN001
Clinical Trials (2)
Total enrollment: 13 patients across 2 trials
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Start: May 2016Est. completion: Oct 20225 patients
Phase 2/3Completed
Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
Start: Apr 2014Est. completion: Oct 20228 patients
Phase 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 companies competing in this space
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