Alexander Disease
0
Pipeline Programs
1
Companies
3
Clinical Trials
1 recruiting
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
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 1 programs with unclassified modality
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ionis PharmaceuticalsCARLSBAD, CA
2 programsEvaluation of Outcome Metrics in Alexander DiseaseN/A1 trial
zilganersenPHASE_3RNA Therapeutic2 trials
Active Trials
NCT07487389Approved For Marketing
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Ionis Pharmaceuticalszilganersen
Ionis PharmaceuticalsEvaluation of Outcome Metrics in Alexander Disease
Clinical Trials (3)
Total enrollment: 254 patients across 3 trials
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
Start: Jun 2021Est. completion: Jul 203454 patients
Phase 3Active Not Recruiting
Zilganersen Expanded Access Program for Individuals With Alexander Disease
N/AApproved For Marketing
Evaluation of Outcome Metrics in Alexander Disease
Start: Jan 2016Est. completion: Dec 2030200 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
1 actively recruiting trials targeting 254 patients
1 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.