Other
Menkes Disease
10
Pipeline Programs↗
2
Active Trials↗
8
Companies Involved↗
Menkes disease is an early-stage landscape with 10 programs across 8 companies. The most active players are Cyprium Therapeutics, Inc., Lundbeck, Sanofi. 6 therapies are approved.
Intelligence
Pipeline Metrics
Competitive Density
Pipeline Velocity
Mech. Concentration
Completion Rate
Attrition Rate
Competition
Competitive Landscape
Bubble size = clinical trials
Approved
Phase 3
Phase 2
Phase 1
Preclinical
Key Players
Top Companies Companies ranked by the number of pipeline programs they run for this disease.
Pipeline
Programs by Phase
Preclinical2
Phase 1
No programs
Phase 3
No programs
Analysis
Trial Status
6
clinical trials
Completed3 · 50%
Approved for marketing1 · 17%
Active, not recruiting1 · 17%
Recruiting1 · 17%
Clinical Trials
Trial Activity
6 trials
Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.
Recruiting·—·Phase 1/2·NCT07398508·6 enrolled
NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
Completed·—·Phase 1/2·NCT04977388·3 enrolled
Copper Histidine Therapy for Menkes Diseases
Completed·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·Phase 1/2·NCT00001262·60 enrolled
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)