Perampanel

Summary

Perampanel (CHEMBL1214124) is an approved small-molecule AMPA receptor antagonist (ATC N03AX22); indicated across 17 conditions including epilepsy and visual epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AX22
• Indications: 17 conditions
• Clinical trials: 100
• Chemistry: 349.4 Da · C23H15N3O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1214124
Name	Perampanel
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9924495
ChEBI	CHEBI:71013
ATC	N03AX22
Molecular formula	C23H15N3O
Molecular weight	349.4
InChIKey	PRMWGUBFXWROHD-UHFFFAOYSA-N

SMILES: C1=CC=C(C=C1)N2C=C(C=C(C2=O)C3=CC=CC=C3C#N)C4=CC=CC=N4

IUPAC name: 2-(2-oxo-1-phenyl-5-pyridin-2-yl-3-pyridinyl)benzonitrile

ChEBI definition: A member of the class of bipyridines that is 2,3’-bipyridin-6’-one substituted at positions 1’ and 5’ by phenyl and 2-cyanophenyl groups respectively. Used as an adjunctive therapy for the treatment of partial-onset seizures in patients with epilepsy.

Pharmacological roles (ChEBI): AMPA receptor antagonist, anticonvulsant.

Also known as: E-2007, E2007, Fycompa, Perampanel, PERAMPANEL, perampanel

Patent coverage: 631 distinct patent families (1,558 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 1,279 (82%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Glutamate receptor 1, 3’,5’-cyclic-AMP phosphodiesterase 4D, Glutamate receptor 3.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 7 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GRIA1	6.61	IC50	243	nM	CHEMBL_ACT_16635706
GRIA1	6.31	IC50	485	nM	CHEMBL_ACT_16635691
GRIA1	6.21	IC50	623	nM	CHEMBL_ACT_16635708
GRIA1	5.93	IC50	1180	nM	CHEMBL_ACT_16635675
GRIA3	5.68	IC50	2100	nM	CHEMBL_ACT_25090888
GRIA1	5.19	IC50	6510	nM	CHEMBL_ACT_16635710

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

17 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
epilepsy	4	MONDO:0005027	EFO:0000474
visual epilepsy	4	MONDO:0001386	HP:0001250
focal epilepsy	4	MONDO:0005384	EFO:0004263
epilepsy with generalized tonic-clonic seizures	3	MONDO:0005754	HP:0002069
Parkinson disease	3	MONDO:0005180	MONDO:0005180
Lennox-Gastaut syndrome	3	MONDO:0016532	MONDO:0016532
essential tremor	2	MONDO:0003233	EFO:0003108
diabetic neuropathy	2	MONDO:0006626	EFO:1000783
neuralgia	2	MONDO:0021667	EFO:0009430
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
migraine disorder	2	MONDO:0005277	MONDO:0005277
major depressive disorder	1	MONDO:0002009	MONDO:0002009
alcohol abuse	1	MONDO:0002046	MONDO:0007079
bipolar disorder	1	MONDO:0004985	MONDO:0004985

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 100.

Phase distribution

Phase	Trials
PHASE2	25
Not specified	22
PHASE3	16
PHASE1	15
PHASE4	14
PHASE1/PHASE2	4
PHASE2/PHASE3	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02220972	PHASE4	WITHDRAWN	To Evaluate the Effect of Perampanel on Objective and Subjective Sleep in Subjects With Insomnia and Partial Onset Seizures
NCT02363933	PHASE4	COMPLETED	Perampanel in Seizure Patients With Primary Glial Brain Tumors
NCT02726074	PHASE4	COMPLETED	Trial Evaluating the Efficacy and Safety of Perampanel Added to Monotherapy in Participants With Partial Onset Seizures With or Without Secondary Generalization
NCT02727101	PHASE4	TERMINATED	Perampanel for Treatment of Adults With Refractory Focal Epilepsy : a Pilot Study.
NCT02900755	PHASE4	COMPLETED	Effects of Perampanel on Cognition and Electroencephalography in Patients With Epilepsy
NCT03288129	PHASE4	COMPLETED	Study to Evaluate the Efficacy and Safety of Perampanel as Monotherapy or First Adjunctive Therapy in Subjects With Partial Onset Seizures With or Without Secondarily Generalized Seizures or With Primary Generalized Tonic-Clonic Seizures
NCT03457129	PHASE4	COMPLETED	Fycompa Titration Intervals and Effects on Retention Rate
NCT03653741	PHASE4	COMPLETED	Effects of Perampanel on Neurophysiology Test Perimeters
NCT04118829	PHASE4	UNKNOWN	Correlation of Serum Level of Perampanel (PER) Level to Cerebrospinal Fluid (CSF) Levels of PER
NCT04417907	PHASE4	TERMINATED	Perampanel Titration and Cognitive Effects
NCT04650204	PHASE4	TERMINATED	Perampanel for the Reduction of Seizure Frequency in Patients With High-grade Glioma and Focal Epilepsy
NCT05201703	PHASE4	TERMINATED	Fycompa in Catamenial Epilepsy
NCT05533814	PHASE4	COMPLETED	A Study to Evaluate the Efficacy and Safety of Perampanel Monotherapy in Untreated Participants With Focal Onset Seizures With or Without Focal to Bilateral Tonic-clonic Seizures
NCT05684978	PHASE4	TERMINATED	Efficacy and Safety of Perampanel in the Treatment of Refractory Status Epilepticus
NCT00286897	PHASE3	COMPLETED	The Efficacy, Safety and Tolerability of E2007 in Levodopa Treated Parkinson’s Disease Patients With Motor Fluctuations
NCT00360308	PHASE3	TERMINATED	Efficacy, Safety and Tolerability of E2007 in Levodopa Treated Parkinson’s Disease Patients With Motor Fluctuations
NCT00360412	PHASE3	TERMINATED	A Study of E2007 as an Adjunctive Therapy in Levodopa Treated Parkinson’s Disease Patients With Motor Fluctuations
NCT00368108	PHASE3	COMPLETED	Efficacy, Safety, and Tolerability of E2007 in Levodopa Treated Parkinson’s Disease Patients With Motor Fluctuations
NCT00505284	PHASE2/PHASE3	COMPLETED	An Evaluation of the Efficacy and Safety of E2007 in Patients With Painful Diabetic Neuropathy
NCT00505622	PHASE3	TERMINATED	Evaluation of the Long-term Safety, Tolerability, and Efficacy of Perampanel (E2007) as an Adjunctive Therapy in Levodopa Treated Parkinson’s Disease Subjects With Motor Fluctuations
NCT00592904	PHASE2/PHASE3	COMPLETED	Evaluating E2007 (Perampanel) in Patients With Painful Diabetic Neuropathy (PDN) or Post-Herpetic Neuralgia (PHN)
NCT00699582	PHASE3	COMPLETED	To Evaluate The Efficacy and Safety of E2007 (Perampanel) Given as Adjunctive Therapy in Subjects With Refractory Partial Seizures
NCT00699972	PHASE3	COMPLETED	Evaluating the Efficacy and Safety of E2007 (Perampanel) Given as Adjunctive Therapy in Subjects With Refractory Partial Seizures
NCT00700310	PHASE3	COMPLETED	Evaluating Efficacy and Safety of E2007 (Perampanel) Given as Adjunctive Therapy in Subjects With Refractory Partial Seizures
NCT00735397	PHASE3	COMPLETED	Evaluation of the Efficacy and Safety of E2007 (Perampanel) Given as Adjunctive Therapy in Subjects With Refractory Partial Seizures
NCT01393743	PHASE3	COMPLETED	A Efficacy and Safety Study of Adjunctive Perampanel in Primary Generalized Tonic Clonic Seizures
NCT01618695	PHASE3	COMPLETED	A Study With an Open-label Extension Phase to Evaluate the Efficacy and Safety of Perampanel (E2007) Administered as an Adjunctive Therapy in Subjects With Refractory Partial-onset Seizures
NCT02427607	PHASE3	COMPLETED	An Open-label Extension Study to Evaluate the Safety and Tolerability of Perampanel (E2007) Administered as an Adjunctive Therapy in Epilepsy Subjects
NCT02834793	PHASE3	TERMINATED	Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome
NCT02849626	PHASE3	COMPLETED	Perampanel as Adjunctive Therapy in Pediatrics With Partial Onset Seizures or Primary Generalized Tonic Clonic Seizures
NCT03201900	PHASE3	COMPLETED	Study for Verification of Efficacy and Safety for Perampanel Monotherapy in Untreated Participants With Partial Onset Seizures (Including Secondarily Generalized Seizures (FREEDOM Study)
NCT04309721	PHASE3	TERMINATED	Perampanel in Focal Status Epilepticus
NCT04015141	PHASE2	RECRUITING	A Study to Evaluate Efficacy and Safety of Perampanel Administered as an Adjunctive Therapy in Pediatric Participants With Childhood Epilepsy
NCT05786066	PHASE2	RECRUITING	The Impact of AMPA Receptor Blockade on Ketamine’s Anti-Suicidal Effects
NCT06401707	PHASE2	RECRUITING	PeRampanel fOr Status ePilEpticus pRophylaxis Post-cardiac Arrest
NCT00144690	PHASE2	COMPLETED	E2007 Given as Adjunctive Therapy in Patients With Refractory Partial Seizures
NCT00154063	PHASE2	COMPLETED	Efficacy and Safety Study of E2007 in Migraine Prophylaxis
NCT00165789	PHASE2	COMPLETED	A Study to Explore the Safety And Tolerability of Doses of E2007 Up to a Maximum of 8 mg In Patients With Parkinson’s Disease Who Experience End-of-Dose Wearing Off Motor Fluctuations
NCT00368472	PHASE2	COMPLETED	4-Year Open-Label Extension Phase of the Parallel-Group Study of E2007 in Patients With Refractory Partial Seizures
NCT00416195	PHASE2	COMPLETED	Exploring the Safety And Tolerability of Doses of E2007 up to a Maximum of 12 mg In Patients With Refractory Partial Seizures

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 5 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).