What is in the research pipeline for Dravet?

Clinical Trial Phases

    • Phase I: Researchers test a new drug or treatment in a small group of healthy volunteers and/or people with the disease for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Purpose: Safety and Dosage
    • Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. Purpose: Efficacy and Side Effects
    • Phase III: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. More than one Phase III study may be required before a New Drug Application (NDA) may be submitted to the FDA. Purpose: Efficacy and Adverse Reactions
    • FDA Approval
    • Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug’s effect in various populations and any side effects associated with long-term use. Purpose: Safety and Efficacy

The following companies have indicated that they are working on a medication or treatment that might be effective for patients living with Dravet syndrome. We have added links to each website for more information.

DSF places a high priority on funding research that has a clear path to genetic understanding, clinical application, and/or therapeutic development. Click here to view DSF Funded Research. 

Interested in participating in research? Find actively enrolling clinical trials for Dravet syndrome here.

Biocodex- Diacomit (stiripentol) – FDA Approved
Jazz Pharmaceuticals (prev. Greenwich BioSciences) – Epidiolex (CBD) – FDA Approved
UCB/Zogenix – Fenfluramine (ZX008) – FDA Approved
Eisai – lorcaserin – Phase 3
Epygenix – EPX-100 (clemizole HCL) – Phase 2
Takeda – TAK935 (soticlestat) – Phase 3
Stoke Therapeutics – TANGO STK-001 – Phase 1/2a
Encoded Therapeutics (AAV-ETX101) – Preclinical
Supernus Pharmaceuticals – SPN-817/BIS-001/Huperzine A – Phase I
CAMP4 – CMP-SCN – Preclinical

 Denotes US FDA Orphan Drug Designation for Dravet syndrome

Updated April 2021