Congenital Myasthenic Syndrome
1
Pipeline Programs
2
Companies
4
Clinical Trials
2 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
argenxBelgium - Zwijnaarde
2 programs1
ARGX-119Phase 11 trial
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations N/A1 trial
Active Trials
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs3,4-DiaminopyridineN/A1 trial
Amifampridine PhosphateN/A1 trial
Active Trials
NCT03062631No Longer Available
NCT02189720Approved For Marketing
Trial Timeline
Clinical trial activity over time
2024
2025
2026
2027
2028
argenxARGX-119
argenxA Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations
Clinical Trials (4)
Total enrollment: 116 patients across 4 trials
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Start: Sep 2024Est. completion: Jan 202816 patients
Phase 1Recruiting
Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia
N/ANo Longer Available
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
N/AApproved For Marketing
NCT06078553argenxA Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Start: Feb 2024Est. completion: Jun 2027100 patients
N/ARecruiting
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
2 actively recruiting trials targeting 116 patients
2 companies competing in this space