Fragile X Syndrome
Rare Diseases
5
Pipeline Programs
4
Companies
6
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
5
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
4 companies ranked by most advanced pipeline stage
Jazz PharmaceuticalsCA - Palo Alto
2 programs2
CBD Oral SolutionPhase 21 trial
CBD Oral SolutionPhase 21 trial
Active Trials
M&
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Mirum PharmaceuticalsLow dose of MRM-3379
Jazz PharmaceuticalsCBD Oral Solution
Jazz PharmaceuticalsCBD Oral Solution
HealxGaboxadol
HealxSulindac
Merck & Co.Behavior analytic treatment
Clinical Trials (6)
Total enrollment: 150 patients across 6 trials
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
Start: Nov 2025Est. completion: Oct 202760 patients
Phase 2Recruiting
Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome
Start: May 2025Est. completion: Dec 202740 patients
Phase 2Not Yet Recruiting
Effect of CBD on the Brain
Start: May 2025Est. completion: Dec 202750 patients
Phase 2Not Yet Recruiting
Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
Start: Jan 2024Est. completion: Jun 2025
Phase 2Completed
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
Start: May 2022Est. completion: Oct 2022
Phase 2Withdrawn
Treatment of Disruptive Behaviors in Fragile X Syndrome
Start: Jul 2016Est. completion: Mar 2022
N/ACompleted
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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 150 patients
4 companies competing in this space