Dravet Syndrome

16
Pipeline Programs
4
Companies
17
Clinical Trials
4 recruiting
2
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
4
1
0
8
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 15 programs with unclassified modality

On Market (2)

Approved therapies currently available

U
FENFLURAMINEApproved
fenfluramine
Unknown Company
oral
UP
FINTEPLAApproved
fenfluramine
UCB Pharma
oral2020

Competitive Landscape

4 companies ranked by most advanced pipeline stage

UP
UCB PharmaBelgium - Brussels
8 programs
1
6
1
ZX008Phase 31 trial
ZX008Phase 31 trial
ZX008Phase 31 trial
ZX008Phase 31 trial
ZX008Phase 31 trial
+3 more programs
Active Trials
NCT03780127Approved For Marketing
NCT03467113Completed9Est. Jun 2020
NCT02926898Completed87Est. Jun 2018
+5 more trials
Stoke Therapeutics
3 programs
1
1
1
zorevunersenPhase 3RNA Therapeutic1 trial
zorevunersenPhase 2RNA Therapeutic1 trial
STK-001 - Single Ascending DosesPhase 1/21 trial
Active Trials
NCT04442295Completed62Est. Nov 2023
NCT04740476Active Not Recruiting60Est. Mar 2029
NCT06872125Recruiting170Est. Oct 2028
Harmony Biosciences
Harmony BiosciencesPLYMOUTH MEETING, PA
1 program
1
Clemizole HClPhase 31 trial
Active Trials
NCT04462770RecruitingEst. May 2029
Encoded Therapeutics
5 programs
3
ETX101Phase 1/21 trial
ETX101Phase 1/21 trial
ETX101Phase 1/21 trial
GABA Blood LevelN/A1 trial
Transcranial Magnetic StimulationN/A1 trial
Active Trials
NCT05651204Recruiting36Est. Sep 2027
NCT04614506Unknown6Est. Dec 2022
NCT06112275Active Not Recruiting4Est. Sep 2030
+2 more trials

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
Stoke Therapeuticszorevunersen
Encoded TherapeuticsETX101
Encoded TherapeuticsETX101
Encoded TherapeuticsETX101
Stoke TherapeuticsSTK-001 - Single Ascending Doses
UCB PharmaZX008 0.2 to 0.8 mg/kg/day
Encoded TherapeuticsGABA Blood Level

Showing 15 of 16 trials with date data

Clinical Trials (17)

Total enrollment: 1,565 patients across 17 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 2029
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

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

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

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
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

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

Phase Legend

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

Key Insights

8 late-stage (Phase 3) programs — potential near-term approvals
4 actively recruiting trials targeting 1,565 patients
4 companies competing in this space