Biomarker & Intervention Discovery

Epilepsy

This page lists 14 druggable gene targets, 47 approved or trial-tested drug candidates and 15 recent clinical trials for Epilepsy, with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

US DALYs429.3KWHO GHE 2021 · all ages
Global DALYs14.37MWHO GHE 2021 · worldwide
US mortality3,951 deaths/yrWHO GHE 2021
Global mortality143.7K deaths/yrWHO GHE 2021 · worldwide
NIH funding$216MFY2025 · RCDC estimate
Funding levelModerate
Spontaneous remission60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by…
Best-intervention remissionNot clearly higher than on-medication rates -- the real gap is behavioral/practice
Gap sizeSmall biological gap; large practice gap
Primary barrierpractice
Full remission profile →

Remission & chronicity

Spontaneous remission
60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by syndrome and duration of freedom)
Best-intervention remission
Not clearly higher than on-medication rates -- the real gap is behavioral/practice
Gap size
Small biological gap; large practice gap
Primary barrier
practice
Only 26-45% of seizure-free patients who are candidates for medication withdrawal ever attempt it; physician and patient reluctance vastly exceeds biological risk

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

GABRA1 133GABRA2 237GABRB3 109GRIN2A 74GRIN2B 238HCN1 33KCNQ2 10KCNQ3 16KCNT1 73MTOR 324SCN1A 90SCN2A 248SCN8A 238SLC6A1 6
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHLORDIAZEPOXIDE ~ modulates Mechanistic
ETIFOXINE ↓ inhibits Mechanistic
DIAZEPAM ~ modulates Mechanistic
METHARBITAL ~ modulates Mechanistic
L838417 ~ modulates Mechanistic
GANAXOLONE ~ modulates Mechanistic
THIAMYLAL SODIUM ~ modulates Mechanistic
EVT 201 ? unclear Mechanistic
ADINAZOLAM ? unclear Mechanistic
ZK-93423 ? unclear Mechanistic
AMOBARBITAL ? unclear Mechanistic
ETHCHLORVYNOL ~ modulates Mechanistic
SECOBARBITAL SODIUM ~ modulates Mechanistic
MEPHOBARBITAL ? unclear Mechanistic
ZOLPIDEM TARTRATE ~ modulates Mechanistic
METHAQUALONE ~ modulates Mechanistic
TOPIRAMATE ~ modulates Mechanistic
PAGOCLONE ~ modulates Mechanistic
THIOCOLCHICOSIDE ? unclear Mechanistic
[3H]AZIETOMIDATE ? unclear Mechanistic
PENTOBARBITAL SODIUM ~ modulates Mechanistic
MIDAZOLAM HYDROCHLORIDE ~ modulates Mechanistic
TRIAZOLAM ~ modulates Mechanistic
BARBITURATE ? unclear Mechanistic
LORAZEPAM ~ modulates Mechanistic
BUTOBARBITAL ? unclear Mechanistic
TETRAHYDRODEOXYCORTICOSTERONE ~ modulates Mechanistic
CLOTIAZEPAM ~ modulates Mechanistic
MEPROBAMATE ↑ activates Mechanistic
[3H]CGS8216 ~ modulates Mechanistic
PROPOFOL ~ modulates Mechanistic
CLOBAZAM ~ modulates Mechanistic
SEVOFLURANE ~ modulates Mechanistic
METHOXYFLURANE ~ modulates Mechanistic
LOREDIPLON ~ modulates Mechanistic
CIPEPOFOL ↑ activates Mechanistic
INDIPLON ~ modulates Mechanistic
PHENOBARBITAL ? unclear Mechanistic
PRAZEPAM ~ modulates Mechanistic
CLORAZEPATE ? unclear Mechanistic

Showing top 40 of 133 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHARBITAL ~ modulates Mechanistic
OCINAPLON ? unclear Mechanistic
THIOPENTAL ? unclear Mechanistic
DIAZEPAM ~ modulates Mechanistic 17.0 nM (Ki)
PENTOBARBITAL SODIUM ~ modulates Mechanistic
FLUDIAZEPAM ? unclear Mechanistic
ADINAZOLAM ? unclear Mechanistic
ADIPIPLON ↑ activates Mechanistic
TOPIRAMATE ~ modulates Mechanistic
CLORAZEPATE DIPOTASSIUM ~ modulates Mechanistic
ENFLURANE ~ modulates Mechanistic
FLUNITRAZEPAM ~ modulates Mechanistic
ETOMIDATE ~ modulates Mechanistic
BUTOBARBITAL ? unclear Mechanistic
CLONAZEPAM ~ modulates Mechanistic
ESZOPICLONE ~ modulates Mechanistic
PHENOBARBITAL ? unclear Mechanistic
MEPROBAMATE ↑ activates Mechanistic
PRIMIDONE ~ modulates Mechanistic
ETIFOXINE ↓ inhibits Mechanistic
FLURAZEPAM ? unclear Mechanistic
TEMAZEPAM ~ modulates Mechanistic
ALPRAZOLAM ~ modulates Mechanistic 12.0 nM (EC50)
OXAZEPAM ~ modulates Mechanistic
SECOBARBITAL ? unclear Mechanistic
LORAZEPAM ~ modulates Mechanistic
PRAZEPAM ~ modulates Mechanistic
HALOTHANE ~ modulates Mechanistic
FLUMAZENIL ~ modulates Mechanistic 0.7943 nM (Ki)
BUTABARBITAL SODIUM ~ modulates Mechanistic
ABP-700 ↑ activates Mechanistic
GANAXOLONE ~ modulates Mechanistic
SECOBARBITAL SODIUM ~ modulates Mechanistic
HEXOBARBITAL ? unclear Mechanistic
ZOPICLONE ~ modulates Mechanistic
DARIGABAT ↑ activates Mechanistic
COCAINE ? unclear Mechanistic
MEPHOBARBITAL ? unclear Mechanistic
TALBUTAL ~ modulates Mechanistic
ACAMPROSATE CALCIUM ~ modulates Mechanistic

Showing top 40 of 237 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHOXYFLURANE ~ modulates Mechanistic
QUAZEPAM ~ modulates Mechanistic
GABOXADOL ↑ activates Mechanistic
CINOLAZEPAM ? unclear Mechanistic
TOPIRAMATE ~ modulates Mechanistic
HALOTHANE ~ modulates Mechanistic
CLONAZEPAM ~ modulates Mechanistic
GLUTETHIMIDE ~ modulates Mechanistic
CHLORDIAZEPOXIDE ~ modulates Mechanistic
PRAZEPAM ~ modulates Mechanistic
PAGOCLONE ~ modulates Mechanistic
TRIAZOLAM ~ modulates Mechanistic
RESEQUINIL ↓ inhibits Mechanistic
ETIFOXINE ↓ inhibits Mechanistic
ESZOPICLONE ~ modulates Mechanistic
MIDAZOLAM HYDROCHLORIDE ~ modulates Mechanistic
PROPOFOL ~ modulates Mechanistic
TEMAZEPAM ~ modulates Mechanistic
ALPRAZOLAM ~ modulates Mechanistic
ESTAZOLAM ~ modulates Mechanistic
FOSPROPOFOL ? unclear Mechanistic
ETHCHLORVYNOL ~ modulates Mechanistic
LORAZEPAM ~ modulates Mechanistic
ADIPIPLON ↑ activates Mechanistic
CLOTIAZEPAM ~ modulates Mechanistic
FLUDIAZEPAM ? unclear Mechanistic
TALBUTAL ~ modulates Mechanistic
METHARBITAL ~ modulates Mechanistic
CHLORMETHIAZOLE ~ modulates Mechanistic
ETAZOLATE ~ modulates Mechanistic
CIPEPOFOL ↑ activates Mechanistic
HALAZEPAM ~ modulates Mechanistic
ACAMPROSATE CALCIUM ~ modulates Mechanistic
CHLORDIAZEPOXIDE HYDROCHLORIDE ~ modulates Mechanistic
FLUMAZENIL ~ modulates Mechanistic
SEVOFLURANE ~ modulates Mechanistic
METHOHEXITAL SODIUM ~ modulates Mechanistic
METHYPRYLON ~ modulates Mechanistic
THIAMYLAL SODIUM ~ modulates Mechanistic
BROMAZEPAM ? unclear Mechanistic

Showing top 40 of 109 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
ORPHENADRINE CITRATE ↓ inhibits Mechanistic
PHILANTHOTOXIN 343 ? unclear Mechanistic
AZD8108 ↓ inhibits Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
CONANTOKIN-G ↓ inhibits Mechanistic
NVP-AAM077 ↓ inhibits Mechanistic
GW468816 ↓ inhibits Mechanistic
APIMOSTINEL ↑ activates Mechanistic
SELFOTEL ↓ inhibits Mechanistic
MEMANTINE HYDROCHLORIDE ~ modulates Mechanistic
FELBAMATE ↓ inhibits Mechanistic
NEBOGLAMINE ~ modulates Mechanistic
ZOLBETUXIMAB ? unclear Mechanistic
GV196771A ↓ inhibits Mechanistic
[3H]CGP61594 ↓ inhibits Mechanistic
[3H]DIZOCILPINE ↓ inhibits Mechanistic
(RS)-(TETRAZOL-5-YL)GLYCINE ↑ activates Mechanistic
L689560 ↓ inhibits Mechanistic
PERZINFOTEL ↓ inhibits Mechanistic
AV-101 ↓ inhibits Mechanistic
GLYCINE ↑ activates Mechanistic
KETAMINE HYDROCHLORIDE ↓ inhibits Mechanistic
MG2+ ↓ inhibits Mechanistic
NERAMEXANE ? unclear Mechanistic
D-AP5 ↓ inhibits Mechanistic
NBQX ? unclear Mechanistic
L701324 ↓ inhibits Mechanistic
LANICEMINE ↓ inhibits Mechanistic
NERAMEXANE MESYLATE ↓ inhibits Mechanistic
CGP37849 ↓ inhibits Mechanistic
ESKETAMINE ~ modulates Mechanistic
INDANTADOL ? unclear Mechanistic
D-CCPENE ↓ inhibits Mechanistic
DEXANABINOL ? unclear Mechanistic
[3H]D-ASPARTIC ACID ↑ activates Mechanistic
TCN-201 ~ modulates Mechanistic
LY233053 ↓ inhibits Mechanistic
SAGE-718 ~ modulates Mechanistic
(+)-HA966 ↑ activates Mechanistic
NMDA ↑ activates Mechanistic

Showing top 40 of 74 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
DIMIRACETAM ? unclear Mechanistic
KETAMINE HYDROCHLORIDE ↓ inhibits Mechanistic
PERZINFOTEL ↓ inhibits Mechanistic
[3H]CGP39653 ↓ inhibits Mechanistic
PHENCYCLIDINE ↓ inhibits Mechanistic
DEXANABINOL ? unclear Mechanistic
RAPASTINEL ↑ activates Mechanistic
DELUCEMINE ↓ inhibits Mechanistic
AMANTADINE HYDROCHLORIDE ↓ inhibits Mechanistic
RISLENEMDAZ ↓ inhibits Mechanistic
ORPHENADRINE CITRATE ↓ inhibits Mechanistic
ELIPRODIL ? unclear Mechanistic 13.0 nM (Ki)
INDANTADOL ? unclear Mechanistic
SEVOFLURANE ? unclear Mechanistic
SAGE-718 ~ modulates Mechanistic
L689560 ↓ inhibits Mechanistic
ONFASPRODIL ~ modulates Mechanistic
HOMOQUINOLINIC ACID ↑ activates Mechanistic
NEBOGLAMINE ~ modulates Mechanistic
DIZOCILPINE ↓ inhibits Mechanistic
LY233053 ↓ inhibits Mechanistic
GLYCINE ↑ activates Mechanistic
ESKETAMINE ~ modulates Mechanistic
[3H]CGP61594 ↓ inhibits Mechanistic
ORPHENADRINE HYDROCHLORIDE ↓ inhibits Mechanistic
[3H]MDL105519 ↓ inhibits Mechanistic
RADIPRODIL ? unclear Mechanistic
NBQX ? unclear Mechanistic
SEMAPIMOD ↓ inhibits Mechanistic
SELFOTEL ↓ inhibits Mechanistic
SERINE, D- ↑ activates Mechanistic
COMPOUND 18C [PMID: 25901531] ↓ inhibits Mechanistic
AZD8108 ↓ inhibits Mechanistic
[3H]L689560 ↓ inhibits Mechanistic
TRAXOPRODIL ↓ inhibits Mechanistic 74.0 nM (IC50)
HALOPERIDOL DECANOATE ? unclear Mechanistic
N1-DANSYL-SPERMINE ↓ inhibits Mechanistic
[3H]D-ASPARTIC ACID ↑ activates Mechanistic
NERAMEXANE ? unclear Mechanistic
NVP-AAM077 ↓ inhibits Mechanistic

Showing top 40 of 238 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
TRAMADOL ? unclear Mechanistic
DESMETRAMADOL ? unclear Mechanistic
CHEMBL2052019 ↓ inhibits Mechanistic 158.49 nM (IC50)
CHEMBL1823665 ↓ inhibits Mechanistic 1258.93 nM (IC50)
CHEMBL1823666 ↓ inhibits Mechanistic 398.11 nM (IC50)
CHEMBL1823667 ↓ inhibits Mechanistic 630.96 nM (IC50)
CHEMBL1823668 ↓ inhibits Mechanistic 25118.86 nM (IC50)
CHEMBL1823669 ↓ inhibits Mechanistic 63095.73 nM (IC50)
CHEMBL1823670 ↓ inhibits Mechanistic 100000.0 nM (IC50)
CHEMBL1823671 ↓ inhibits Mechanistic 19952.62 nM (IC50)
CHEMBL1823673 ↓ inhibits Mechanistic 25118.86 nM (IC50)
CHEMBL1823674 ↓ inhibits Mechanistic 630.96 nM (IC50)
CHEMBL1823675 ↓ inhibits Mechanistic 50118.72 nM (IC50)
CHEMBL1823676 ↓ inhibits Mechanistic 15848.93 nM (IC50)
CHEMBL1435630 ↓ inhibits Mechanistic 398.11 nM (IC50)
CHEMBL1823677 ↓ inhibits Mechanistic 125.89 nM (IC50)
CHEMBL1823678 ↓ inhibits Mechanistic 63095.73 nM (IC50)
CHEMBL1823874 ↓ inhibits Mechanistic 15.85 nM (IC50)
CHEMBL1823875 ↓ inhibits Mechanistic 31.62 nM (IC50)
CHEMBL1823876 ↓ inhibits Mechanistic 251.19 nM (IC50)
CHEMBL1823878 ↓ inhibits Mechanistic 31.62 nM (IC50)
CHEMBL1823879 ↓ inhibits Mechanistic 50.12 nM (IC50)
CHEMBL1823880 ↓ inhibits Mechanistic 79.43 nM (IC50)
CHEMBL1823881 ↓ inhibits Mechanistic 199.53 nM (IC50)
CHEMBL1823882 ↓ inhibits Mechanistic 398.11 nM (IC50)
CHEMBL1823883 ↓ inhibits Mechanistic 15848.93 nM (IC50)
CHEMBL1823885 ↓ inhibits Mechanistic 1584.89 nM (IC50)
CHEMBL1823886 ↓ inhibits Mechanistic 398.11 nM (IC50)
CHEMBL1823887 ↓ inhibits Mechanistic 316.23 nM (IC50)
CHEMBL1823888 ↓ inhibits Mechanistic 630.96 nM (IC50)
CHEMBL1823889 ↓ inhibits Mechanistic 2511.89 nM (IC50)
CHEMBL6195028 ↓ inhibits Mechanistic 48.0 nM (IC50)
CHEMBL6195037 ↓ inhibits Mechanistic 40000.0 nM (IC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
GUANIDINE HYDROCHLORIDE ↓ inhibits Mechanistic
TEDISAMIL ↓ inhibits Mechanistic
DALFAMPRIDINE ↓ inhibits Mechanistic
EZOGABINE ↑ activates Mechanistic
AMIFAMPRIDINE PHOSPHATE ↓ inhibits Mechanistic
NERISPIRDINE ↓ inhibits Mechanistic
ICA-105665 ? unclear Mechanistic
FLINDOKALNER ↑ activates Mechanistic
CELECOXIB ? unclear Mechanistic
AMIFAMPRIDINE ↓ inhibits Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EZOGABINE ↑ activates Mechanistic 3900.0 nM (EC50)
AZETUKALNER ↑ activates Mechanistic
DALFAMPRIDINE ↓ inhibits Mechanistic
NERISPIRDINE ↓ inhibits Mechanistic
ICA-105665 ? unclear Mechanistic
GUANIDINE HYDROCHLORIDE ↓ inhibits Mechanistic
CELECOXIB ? unclear Mechanistic
GABAPENTIN ↑ activates Mechanistic
LINOPIRDINE ? unclear Mechanistic
FLINDOKALNER ↑ activates Mechanistic 2400.0 nM (EC50)
TETRAMETHYLAMMONIUM ↓ inhibits Mechanistic
AMIFAMPRIDINE PHOSPHATE ↓ inhibits Mechanistic
TEDISAMIL ↓ inhibits Mechanistic
AMIFAMPRIDINE ↓ inhibits Mechanistic
CHEMBL4545892 ↑ activates Mechanistic 10500.0 nM (EC50)
CHEMBL4457044 ↑ activates Mechanistic 920.0 nM (EC50)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PHORBOL 12-MYRISTATE 13-ACETATE ? unclear Mechanistic
NICLOSAMIDE ↑ activates Mechanistic
QUINIDINE ↓ inhibits Mechanistic 15800.0 nM (IC50)
LOXAPINE ↑ activates Mechanistic
CLOFILIUM ↓ inhibits Mechanistic
CHEMBL4793291 ↓ inhibits Mechanistic 167.0 nM (IC50)
CHEMBL4763819 ↓ inhibits Mechanistic 3314.0 nM (IC50)
CHEMBL4793050 ↓ inhibits Mechanistic 11430.0 nM (IC50)
CHEMBL4783466 ↓ inhibits Mechanistic 335.0 nM (IC50)
CHEMBL4789512 ↓ inhibits Mechanistic 1597.0 nM (IC50)
CHEMBL4779088 ↓ inhibits Mechanistic 977.0 nM (IC50)
CHEMBL4783741 ↓ inhibits Mechanistic 136.0 nM (IC50)
CHEMBL4759833 ↓ inhibits Mechanistic 6489.0 nM (IC50)
CHEMBL4750826 ↓ inhibits Mechanistic 1373.0 nM (IC50)
CHEMBL4777757 ↓ inhibits Mechanistic 4466.0 nM (IC50)
CHEMBL4786255 ↓ inhibits Mechanistic 165.0 nM (IC50)
CHEMBL4751954 ↓ inhibits Mechanistic 5245.0 nM (IC50)
CHEMBL4755742 ↓ inhibits Mechanistic 11800.0 nM (IC50)
CHEMBL4744857 ↓ inhibits Mechanistic 1410.0 nM (IC50)
CHEMBL4740667 ↓ inhibits Mechanistic 2642.0 nM (IC50)
CHEMBL4788669 ↓ inhibits Mechanistic 27.0 nM (IC50)
CHEMBL4781553 ↓ inhibits Mechanistic 573.0 nM (IC50)
CHEMBL4795194 ↓ inhibits Mechanistic 454.0 nM (IC50)
CHEMBL4743249 ↓ inhibits Mechanistic 505.0 nM (IC50)
CHEMBL4763955 ↓ inhibits Mechanistic 16.0 nM (IC50)
CHEMBL4750925 ↓ inhibits Mechanistic 322.0 nM (IC50)
CHEMBL4754084 ↓ inhibits Mechanistic 61.0 nM (IC50)
CHEMBL4793279 ↓ inhibits Mechanistic 195.0 nM (IC50)
CHEMBL4741793 ↓ inhibits Mechanistic 40.0 nM (IC50)
CHEMBL4776478 ↓ inhibits Mechanistic 6282.0 nM (IC50)
CHEMBL4745211 ↓ inhibits Mechanistic 744.0 nM (IC50)
CHEMBL4758890 ↓ inhibits Mechanistic 778.0 nM (IC50)
CHEMBL4744290 ↓ inhibits Mechanistic 9285.0 nM (IC50)
CHEMBL4750807 ↓ inhibits Mechanistic 1876.0 nM (IC50)
CHEMBL5218502 ↓ inhibits Mechanistic 3200.0 nM (IC50)
CHEMBL5219121 ↓ inhibits Mechanistic 3000.0 nM (IC50)
CHEMBL1730394 ↓ inhibits Mechanistic 2400.0 nM (IC50)
CHEMBL5219965 ↓ inhibits Mechanistic 4900.0 nM (IC50)
CHEMBL5218475 ↓ inhibits Mechanistic 1400.0 nM (IC50)
CHEMBL5220721 ↓ inhibits Mechanistic 2400.0 nM (IC50)

Showing top 40 of 73 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
VISTUSERTIB ↓ inhibits Mechanistic
EVEROLIMUS ↓ inhibits Mechanistic
SIROLIMUS ↓ inhibits Mechanistic 3.467 nM (IC50)
PYRROLIDINE DITHIOCARBAMATE ? unclear Mechanistic
SU9516 ? unclear Mechanistic
THAPSIGARGIN ? unclear Mechanistic
CGS 15943 ? unclear Mechanistic
RIDAFOROLIMUS ↓ inhibits Mechanistic
PALOMID-529 ↓ inhibits Mechanistic
PROPAFENONE ? unclear Mechanistic
ZOTAROLIMUS ? unclear Mechanistic 3.3 nM (IC50)
GEDATOLISIB ↓ inhibits Mechanistic
TEMSIROLIMUS ↓ inhibits Mechanistic
DS-3078A ↓ inhibits Mechanistic
MTOR INHIBITOR ? unclear Mechanistic
SYK INHIBITOR III ? unclear Mechanistic
CHEMBL:CHEMBL587620 ? unclear Mechanistic
PF-04691502 ↓ inhibits Mechanistic
LY-294002 ? unclear Mechanistic 2500.0 nM (IC50)
ISOPROTERENOL ? unclear Mechanistic
ME-344 ? unclear Mechanistic
ALPELISIB ↓ inhibits Mechanistic
SAMOTOLISIB ↓ inhibits Mechanistic
PERPHENAZINE ? unclear Mechanistic
MIBEFRADIL ? unclear Mechanistic
TG100-115 ? unclear Mechanistic
TAFA-93 ? unclear Mechanistic
SF1126 ↓ inhibits Mechanistic
SIROLIMUS ALBUMIN-BOUND NANOPARTICLES ? unclear Mechanistic
GOSSYPOL ? unclear Mechanistic
PHENETHYLISOTHIOCYANATE ? unclear Mechanistic
CHEMBL:CHEMBL599088 ? unclear Mechanistic
CHEMBL:CHEMBL600334 ? unclear Mechanistic
CHEMBL:CHEMBL591618 ? unclear Mechanistic
CAFFEIC ACID PHENETHYL ESTER ? unclear Mechanistic
DIPHENYLENEIODONIUM ? unclear Mechanistic
CHEMBL:CHEMBL605077 ? unclear Mechanistic
VANOXERINE ? unclear Mechanistic
WIN55212-2 ? unclear Mechanistic
SAPANISERTIB ↓ inhibits Mechanistic

Showing top 40 of 324 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
TETRACAINE ↓ inhibits Mechanistic
PHENACEMIDE ↓ inhibits Mechanistic
RILUZOLE ↓ inhibits Mechanistic
SAFINAMIDE ? unclear Mechanistic
PHENYTOIN SODIUM ↓ inhibits Mechanistic
MEPIVACAINE HYDROCHLORIDE ↓ inhibits Mechanistic
CHLOROPROCAINE ↓ inhibits Mechanistic
ESLICARBAZEPINE ACETATE ↓ inhibits Mechanistic
FLUOROURACIL ? unclear Mechanistic
LIDOCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
ETHOSUXIMIDE ? unclear Mechanistic
FOSPHENYTOIN ↓ inhibits Mechanistic
DISOPYRAMIDE PHOSPHATE ↓ inhibits Mechanistic
RUFINAMIDE ↓ inhibits Mechanistic
IRAMPANEL ↓ inhibits Mechanistic
TOPIRAMATE ↓ inhibits Mechanistic
QUINIDINE SULFATE ↓ inhibits Mechanistic
ETIDOCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
INDECAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
DYCLONINE HYDROCHLORIDE ↓ inhibits Mechanistic
QUINIDINE POLYGALACTURONATE ↓ inhibits Mechanistic
ZONISAMIDE ↓ inhibits Mechanistic
LIDOCAINE ↓ inhibits Mechanistic
COCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
NITRAZEPAM ? unclear Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
HEXYLCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
ORPHENADRINE CITRATE ↓ inhibits Mechanistic
PRILOCAINE ↓ inhibits Mechanistic
ENCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
QUINIDINE GLUCONATE ↓ inhibits Mechanistic
CLOBAZAM ? unclear Mechanistic
TOCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
PRIMIDONE ↓ inhibits Mechanistic
ANTICONVULSANT AGENT ? unclear Mechanistic
TETRODOTOXIN ? unclear Mechanistic
MERETHOXYLLINE PROCAINE ↓ inhibits Mechanistic
PROPAFENONE HYDROCHLORIDE ↓ inhibits Mechanistic
CENOBAMATE ↓ inhibits Mechanistic
LACOSAMIDE ↓ inhibits Mechanistic

Showing top 40 of 90 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PROCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
PROPOFOL ? unclear Mechanistic
OXCARBAZEPINE ↓ inhibits Mechanistic
SAFINAMIDE ? unclear Mechanistic
PROCAINAMIDE HYDROCHLORIDE ↓ inhibits Mechanistic
PROPOXYCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
MERETHOXYLLINE PROCAINE ↓ inhibits Mechanistic
DISOPYRAMIDE PHOSPHATE ↓ inhibits Mechanistic
TOCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
TOPIRAMATE ↓ inhibits Mechanistic
PRILOCAINE ↓ inhibits Mechanistic
CENOBAMATE ↓ inhibits Mechanistic
NKTR-171 ↓ inhibits Mechanistic
PHENYTOIN SODIUM ↓ inhibits Mechanistic
QUINIDINE SULFATE ↓ inhibits Mechanistic
TETRACAINE ↓ inhibits Mechanistic 1100.0 nM (IC50)
DRONEDARONE HYDROCHLORIDE ↓ inhibits Mechanistic
PHENAZOPYRIDINE HYDROCHLORIDE ↓ inhibits Mechanistic
FOSPROPOFOL ? unclear Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
ESLICARBAZEPINE ACETATE ↓ inhibits Mechanistic
LIDOCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
MEPIVACAINE HYDROCHLORIDE ↓ inhibits Mechanistic
ORPHENADRINE HYDROCHLORIDE ↓ inhibits Mechanistic
LAMOTRIGINE ↓ inhibits Mechanistic 10000.0 nM (IC50)
DYCLONINE HYDROCHLORIDE ↓ inhibits Mechanistic
TETRODOTOXIN ? unclear Mechanistic 14.0 nM (IC50)
MEXILETINE HYDROCHLORIDE ↓ inhibits Mechanistic
ZONISAMIDE ↓ inhibits Mechanistic
PHENOBARBITAL ? unclear Mechanistic
LACOSAMIDE ↓ inhibits Mechanistic
IRAMPANEL ↓ inhibits Mechanistic
FOSPHENYTOIN ↓ inhibits Mechanistic
MORICIZINE HYDROCHLORIDE ↓ inhibits Mechanistic
RILUZOLE ↓ inhibits Mechanistic 2200.0 nM (IC50)
PHENACEMIDE ↓ inhibits Mechanistic
PROPAFENONE HYDROCHLORIDE ↓ inhibits Mechanistic
ORPHENADRINE CITRATE ↓ inhibits Mechanistic
PRIMIDONE ↓ inhibits Mechanistic
ENCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic

Showing top 40 of 248 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHLOROPROCAINE ↓ inhibits Mechanistic
PRILOCAINE ↓ inhibits Mechanistic
PHENYTOIN SODIUM ↓ inhibits Mechanistic
LACOSAMIDE ↓ inhibits Mechanistic
LIDOCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
RALFINAMIDE ↓ inhibits Mechanistic
ENCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
LIDOCAINE ↓ inhibits Mechanistic
FOSPHENYTOIN ↓ inhibits Mechanistic
CARBAMAZEPINE ↓ inhibits Mechanistic
QUINIDINE POLYGALACTURONATE ↓ inhibits Mechanistic
IRAMPANEL ↓ inhibits Mechanistic
ZANDATRIGINE ? unclear Mechanistic
TOCAINIDE HYDROCHLORIDE ↓ inhibits Mechanistic
TETRACAINE ↓ inhibits Mechanistic 520.0 nM (IC50)
MORICIZINE HYDROCHLORIDE ↓ inhibits Mechanistic
SAFINAMIDE ? unclear Mechanistic
ORPHENADRINE CITRATE ↓ inhibits Mechanistic
NKTR-171 ↓ inhibits Mechanistic
PRIMIDONE ↓ inhibits Mechanistic
TOPIRAMATE ↓ inhibits Mechanistic
MERETHOXYLLINE PROCAINE ↓ inhibits Mechanistic
ROPIVACAINE HYDROCHLORIDE ↓ inhibits Mechanistic
BENOXINATE HYDROCHLORIDE ↓ inhibits Mechanistic
EVENAMIDE ↓ inhibits Mechanistic
HEXYLCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
ARTICAINE HYDROCHLORIDE ↓ inhibits Mechanistic
QUINIDINE GLUCONATE ↓ inhibits Mechanistic
NERISPIRDINE ↓ inhibits Mechanistic
MEPIVACAINE HYDROCHLORIDE ↓ inhibits Mechanistic
ETIDOCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
CENOBAMATE ↓ inhibits Mechanistic
OXCARBAZEPINE ↓ inhibits Mechanistic
PHENAZOPYRIDINE HYDROCHLORIDE ↓ inhibits Mechanistic
MEXILETINE HYDROCHLORIDE ↓ inhibits Mechanistic
LAMOTRIGINE ↓ inhibits Mechanistic
RUFINAMIDE ↓ inhibits Mechanistic
COCAINE HYDROCHLORIDE ↓ inhibits Mechanistic
DYCLONINE HYDROCHLORIDE ↓ inhibits Mechanistic
QUINIDINE SULFATE ↓ inhibits Mechanistic

Showing top 40 of 238 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CI-966 ? unclear Mechanistic
METADOXINE ? unclear Mechanistic
COCAINE ? unclear Mechanistic
TIAGABINE HYDROCHLORIDE ↓ inhibits Mechanistic
HINOKININ ? unclear Mechanistic
TIAGABINE ? unclear Mechanistic

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Epilepsy: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT07125261 Phase: 1 Started: 2025-10-07

This is an open label study to assess the biological effect of BHV-7000 on abnormal activity recorded by the RNS System in patients with focal epilepsy implanted with the RNS System. BHV-7000 is a potassium channel activator being evaluated for use in epilepsy. Participants are offered the drug for ...

NCT05727943 Phase: 2 Started: 2022-03-01

In this exploratory trial, the potential anti-seizure activity of clioquinol in a small cohort of adolescents with drug-resistant epilepsy will be examined. Subjects will be exposed to clioquinol add-on for a period of maximum 8 weeks (2 weeks low dose, 6 weeks higher dose). The main hypothesis of t...

NCT05076838 Phase: 1/2 Started: 2021-11-08

This is a Phase 1/2a, open-label, single-dose, PK study of VALTOCO with open-label safety period in pediatric epilepsy subjects age 2 to 5 years.

NCT02655198 Phase: 2 Started: 2016-01

In this trial, the potential anti-epileptic effect of low dose fenfluramine in Lennox Gastaut epilepsy will be studied. An exploratory dose finding add-on trial is proposed. At baseline and at the end of the study, ECG and ultrasound of the heart will be performed as part of the safety follow up.

NCT05067634 Phase: 3 Started: 2022-01-10

Primary objective: To evaluate the safety and tolerability of cenobamate in pediatric subjects 2-17 years of age with partial-onset (focal) seizures

NCT07600736 Phase: 2 Started: 2026-05-18

This trial will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of ABS-1230 compared with placebo in participants with KCNT1-related epilepsy

NCT05803434 Phase: 2 Started: 2024-03-14

This is a pilot, open-label, phase II study. The main objective of the study is to demonstrate that Cannabidiol (CBD), used in addition to current anti-seizure medications (ASMs) reduces the number and/or severity of motor (generalized, focal, or both) seizures in children and young adults with rare...

NCT05439876 Phase: 2/3 Started: 2022-07-18

The purpose of this study is to evaluate the effect of melatonin for improving sleep in pediatric epilepsy.

NCT07594158 Phase: 3 Started: 2026-06-10

Primary objective: To evaluate the safety and tolerability of cenobamate in Japanese pediatric subjects 2-17 years of age with partial-onset (focal) seizures

NCT07723963 Phase: 2/3 Started: 2026-09-18

This study is being conducted to evaluate the safety and efficacy of JZP926 capsule in participants with Juvenile Myoclonic Epilepsy (JME).

NCT07318870 Phase: 2 Started: 2026-08-24

ProbCluster trial aims at making the proof of concept of antiseizure efficacy of the old antigout PBN in SC, in which Panx1 involvement is relevant on SC pathophysiology and in which efficient therapies are still lacking. The trial will be performed on patients with focal epilepsy in presurgical eva...

NCT06132893 Phase: 2/3 Started: 2024-03-14

The purpose of this study is to determine whether BHV-7000 is effective in the treatment of refractory focal epilepsy.

NCT05232630 Phase: 4 Started: 2022-10-20

This study is a pilot non-controlled clinical trial with adjunctive fenfluramine for the treatment of five different types of developmental and epileptic encephalopathies (DEEs) focused on epileptic and "non-epileptic outcomes": SYNGAP1 and STXBP1 encephalopathies, inv-dup(15) encephalopathy, multif...

NCT06769659 Phase: 2 Started: 2025-03-11

This study will compare the effect of NPT 2042 and placebo in subjects with GGE on the frequency and duration of electroencephalographic absence seizures, separated by a 14-day washout period. The study will be a single-center, double-blind, crossover study with subjects receiving either NPT 2042 BI...

NCT03650452 Phase: 2 Started: 2018-08-08

The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.


Agents Found By Disease-Level Search

Searched directly from “Epilepsy” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 14-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

47 agents shown (approved or with trial evidence) — 39 not found by the gene-target search above.

Lacosamide

Approved · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT02477839 NCT01484977 NCT00655551

Levetiracetam

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06393309 NCT00643500

topiramate

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00236730 NCT00236756

Brivaracetam 2.5 mgNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00464269 NCT00464269

Vitamin ENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00004637

Rufinamide

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01405053

Any other approved Antiepileptic DrugNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01405053

Oral prednisoloneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01575639

EEGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06934356

Eye TrackingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06934356

fMRINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06934356

Ganaxolone

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04391569

Oxcarbazepine

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00618046

ETX101NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06112275

Lyrica (pregabalin)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00351611

Perampanel 10 MGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06450236

Fycompa 10Mg TabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06450236

almotriptan malateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00210509

Mandalas of EmotionsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04220333

Vitamin DNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07085598

NIRSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02899832

ZX008 0.2 or 0.8 mg/kg/dayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03355209

MelatoninNOT in gene-target list

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05439876

ReACTNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02801136

Supportive TherapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02801136

Air polishing with erythritol powderNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06144034

Conventional treatmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06144034

Sodium valproateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06393309

STARNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01851057

Activity Tracker GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04357912

DIXI Medical Microdeep® Micro- Macro Depth electrodesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06105645

Standard electrodesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06105645

Brivaracetam (ucb34714)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00426673

EEG/fMRINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03582345

nL-ATN1-002NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07084311

NeuroPort micro-electrode arrayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02932839

TLE Arm 1NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00486239

Lamotrigine tabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00605371

Lamotrigine capletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00605371

retigabine/ezogabineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01587339

zonisamide

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01587339

pregabalinNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01587339

eslicarbazepine

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01587339

SPN-817NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06798896

Dimension-Based Statistical LearningNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05209386

Computer based task with EEGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06877260

Brivaracetam 10 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00464269

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.