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ZYN002 -

Phase 3

Fragile X Syndrome | Small molecule | Other |Harmony Biosciences Holdings, Inc.|Last Updated: Dec 17, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment215
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04977986Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X SyndromePHASE3 COMPLETED 215Sep 13, 2021Aug 29, 2025Dec 17, 202527 United States, Australia +3
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Study Endpoints
Primary Endpoints
Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 1 score in patients with complete methylation of the FMR1 gene.
Change from Baseline to Week 18

The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials

Secondary Endpoints
Aberrant Behavior Checklist-Community, Fragile X Factor Structure (ABC-C FXS) Pre-specified Subscale 2 in patients with complete methylation of the FMR1 gene.
Change from Baseline to Week 18
Change on the Caregiver Global Impression of Change (CaGI-C) for Pre-specified parameter among patients with complete methylation of the FMR1 gene.
Change from Baseline to Week 18
Clinical Global Impression- Improvement (CGI-I) scale among patients with complete methylation of the FMR1 gene.
Change from Baseline to Week 18
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ZYN002 - transdermal gelEXPERIMENTALPharmaceutically manufactured. Cannabidiol is formulated as a clear gel for transdermal delivery.
Placebo transdermal gelPLACEBO_COMPARATORPlacebo is formulated as a clear gel for transdermal delivery.
Interventions
NameTypeDescription
ZYN002 - transdermal gelDRUGPharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)
PlaceboDRUGPlacebo formulated as a clear gel that can be applied to the skin (transdermal delivery) Other Names: Placebo Comparator Matching Placebo
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Eligibility Criteria
Age Range3 Years — 29 Years
SexALL
Healthy VolunteersNo
Study Sites27

Inclusion Criteria: * Male or female children and adolescents aged 3 to \< 30 years, at the time of Screening. * Patient resides with caregiver who will continue to provide consistent care throughout the study. * Judged by the Investigator to be in generally good health at Screening based upon the ...

Countries:United StatesAustraliaIrelandNew ZealandUnited Kingdom
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