Myasthenic Syndromes, Congenital

2
Pipeline Programs
1
Companies
1
Clinical Trials
1
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
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Catalyst Pharmaceuticals
FIRDAPSEApproved
amifampridine phosphate
Catalyst Pharmaceuticals
oral2018

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
1 program
1
1
amifampridine phosphatePhase 31 trial
Active Trials
NCT02562066CompletedEst. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Catalyst Pharmaceuticalsamifampridine phosphate

Clinical Trials (1)

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

Start: Jan 2016Est. completion: Oct 2019
Phase 3Completed

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

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