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Dravet Syndrome

5
Pipeline Programs
9
Companies
25
Clinical Trials
5 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
0
0
2
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 22 programs with unclassified modality

On Market (1)

Approved therapies currently available

Lundbeck
ONFIApproved
clobazam
Lundbeck
Benzodiazepine [EPC]oral2012
32M Part D

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Lundbeck
LundbeckDenmark - Copenhagen
2 programs
2
1
ClobazamPhase 31 trial
ClobazamPhase 31 trial
Active Trials
NCT02174094Withdrawn0Est. Aug 2015
NCT02187809Terminated3Est. Oct 2015
Ionis Pharmaceuticals
1 program
1
ION337Phase 1/21 trial
Active Trials
NCT07531745Recruiting32Est. Dec 2030
Biocodex
BiocodexFrance - Gentilly
1 program
1
Stiripentol capsulePhase 11 trial
Active Trials
NCT07176832Completed24Est. Aug 2025
UCB Pharma
UCB PharmaBelgium - Brussels
8 programs
Fenfluramine HydrochlorideN/A1 trial
ZX008 0.2 to 0.8 mg/kg/dayPHASE_11 trial
ZX008PHASE_31 trial
ZX008PHASE_31 trial
ZX008PHASE_31 trial
+3 more programs
Active Trials
NCT03780127Approved For Marketing
NCT03467113Completed9Est. Jun 2020
NCT03299842Terminated5Est. Jul 2018
+6 more trials
Encoded Therapeutics
5 programs
GABA Blood LevelN/A1 trial
Transcranial Magnetic StimulationN/A1 trial
ETX101PHASE_1_21 trial
ETX101PHASE_1_21 trial
ETX101PHASE_1_21 trial
Active Trials
NCT05651204Recruiting36Est. Sep 2027
NCT04614506Unknown6Est. Dec 2022
NCT06112275Active Not Recruiting4Est. Sep 2030
+2 more trials
Stoke Therapeutics
3 programs
STK-001 - Single Ascending DosesPHASE_1_21 trial
zorevunersenPHASE_2RNA Therapeutic1 trial
zorevunersenPHASE_3RNA Therapeutic1 trial
Active Trials
NCT04442295Completed62Est. Nov 2023
NCT04740476Active Not Recruiting60Est. Mar 2029
NCT06872125Recruiting170Est. Oct 2028
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
2 programs
Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet PopulationN/A1 trial
StiripentolN/A1 trial
Active Trials
NCT02229032Completed19Est. Jul 2017
NCT01835314No Longer Available
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
FenfluramineN/A1 trial
Active Trials
NCT04437004Approved For Marketing
Harmony Biosciences
Harmony BiosciencesPLYMOUTH MEETING, PA
1 program
Clemizole HClPHASE_31 trial
Active Trials
NCT04462770Recruiting150Est. May 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Stoke Therapeuticszorevunersen
UCB Pharmafenfluramine
Harmony BiosciencesClemizole HCl
UCB PharmaZX008
UCB PharmaZX008
UCB PharmaZX008
UCB PharmaZX008
UCB PharmaZX008
LundbeckClobazam
LundbeckClobazam
Stoke Therapeuticszorevunersen
Ionis PharmaceuticalsION337
Encoded TherapeuticsETX101
Encoded TherapeuticsETX101
Encoded TherapeuticsETX101

Showing 15 of 22 trials with date data

Clinical Trials (25)

Total enrollment: 1,813 patients across 25 trials

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

Start: Jun 2025Est. completion: Oct 2028170 patients
Phase 3Recruiting

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

Start: May 2024Est. completion: Dec 202625 patients
Phase 3Active Not Recruiting

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

Start: Sep 2020Est. completion: May 2029150 patients
Phase 3Recruiting

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Start: Apr 2019Est. completion: May 2025412 patients
Phase 3Completed

A Study to Assess the Usability of the Embrace Seizure Detection Watch in Children and Young Adults With Dravet Syndrome

Start: Jul 2017Est. completion: Jul 20185 patients
Phase 3Terminated

A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome

Start: Jan 2017Est. completion: Jun 201887 patients
Phase 3Completed

An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome

Start: Jun 2016Est. completion: Jan 2023375 patients
Phase 3Completed

A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome

Start: Jan 2016Est. completion: Jul 2020262 patients
Phase 3Completed

Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

Start: Mar 2015Est. completion: Aug 20150
Phase 3Withdrawn

Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome

Start: Mar 2015Est. completion: Oct 20153 patients
Phase 3Terminated

An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Start: Jan 2021Est. completion: Mar 202960 patients
Phase 2Active Not Recruiting

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

Start: Apr 2026Est. completion: Dec 203032 patients
Phase 1/2Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

Start: May 2024Est. completion: Jan 203347 patients
Phase 1/2Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

Start: May 2024Est. completion: Oct 20305 patients
Phase 1/2Active Not Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)

Start: Feb 2024Est. completion: Sep 20304 patients
Phase 1/2Active Not Recruiting
NCT04442295Stoke TherapeuticsSTK-001 - Single Ascending Doses

An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

Start: Jun 2020Est. completion: Nov 202362 patients
Phase 1/2Completed
NCT07176832BiocodexStiripentol capsule

Bioavailability of Stiripentol After Single Oral Dose of Capsule vs Suspension in Healthy Subjects (STILIQ)

Start: Jun 2025Est. completion: Aug 202524 patients
Phase 1Completed
NCT03467113UCB PharmaZX008 0.2 to 0.8 mg/kg/day

A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol

Start: Jan 2018Est. completion: Jun 20209 patients
Phase 1Completed
NCT03780127UCB PharmaFenfluramine Hydrochloride

ZX008 Expanded Access Protocol

N/AApproved For Marketing

Compassionate Use of Stiripentol in Dravet Syndrome

N/ANo Longer Available

Treatment of Dravet Syndrome With Fenfluramine (Expanded Access Protocol)

N/AApproved For Marketing

EEG Dynamics in Lennox-Gastaut Syndrome Patients Undergoing Fenfluramine Treatment

Start: Apr 2026Est. completion: Apr 202820 patients
N/AEnrolling By Invitation

GABA Biomarkers in Dravet Syndrome

Start: Sep 2022Est. completion: Sep 202736 patients
N/ARecruiting
NCT04614506Encoded TherapeuticsTranscranial Magnetic Stimulation

Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome

Start: Nov 2020Est. completion: Dec 20226 patients
N/AUnknown
NCT02229032Colorado TherapeuticsGenetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population

Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population

Start: Aug 2014Est. completion: Jul 201719 patients
N/ACompleted

Related Jobs

Phase Legend

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

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
5 actively recruiting trials targeting 1,813 patients
9 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.