CDKL5 Deficiency Disorder
2
Pipeline Programs
2
Companies
2
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
ZTALMYApproved
ganaxolone
Marinus PharmaceuticalsNeuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator [EPC]oral2022
953K Part D
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
UCB PharmaZX008
Marinus Pharmaceuticalsganaxolone
Clinical Trials (2)
Total enrollment: 188 patients across 2 trials
A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.
Start: Mar 2022Est. completion: Nov 202787 patients
Phase 3Active Not Recruiting
Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
Start: Jun 2018Est. completion: May 2021101 patients
Phase 3Completed
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 late-stage (Phase 3) programs, potential near-term approvals
2 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.