Rufinamide

Summary

Rufinamide (CHEMBL1201754) is an approved small molecule (ATC N03AF03); indicated across 4 conditions including visual epilepsy and epilepsy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N03AF03
• Indications: 4 conditions
• Clinical trials: 12
• Chemistry: 238.19 Da · C10H8F2N4O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201754
Name	Rufinamide
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	129228
ATC	N03AF03
Molecular formula	C10H8F2N4O
Molecular weight	238.19
InChIKey	POGQSBRIGCQNEG-UHFFFAOYSA-N

SMILES: C1=CC(=C(C(=C1)F)CN2C=C(N=N2)C(=O)N)F

IUPAC name: 1-[(2,6-difluorophenyl)methyl]triazole-4-carboxamide

Also known as: 60231/4, Banzel, CGP 33101, CGP-33101, E-2080, E2080, Inovelon, RUF 331, RUF-331, Rufinamida, Rufinamide, SID29217641

Patent coverage: 847 distinct patent families (3,052 SureChEMBL compound mentions), from 1 matched compound structure(s). 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: 1 (assay-derived). Sample: Carbonic anhydrase 5A, mitochondrial.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
CA5A	6.46	Ki	343.8	nM	CHEMBL_ACT_19073674

Target pathways

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

Indications & clinical

Indications

4 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
visual epilepsy	4	MONDO:0001386	HP:0001250
epilepsy	4	MONDO:0005027	EFO:0000474
Lennox-Gastaut syndrome	4	MONDO:0016532	MONDO:0016532

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

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE3	6
Not specified	3
PHASE1	2
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00334958	PHASE3	COMPLETED	Rufinamide Given as Adjunctive Therapy in Participants With Refractory Partial Seizures
NCT00448539	PHASE3	TERMINATED	Open-Label Extension Study of Rufinamide Given as Adjunctive Therapy in Patients With Refractory Partial Seizures
NCT01146951	PHASE3	COMPLETED	A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients (Study E2080-J081-304)
NCT01151540	PHASE3	COMPLETED	A Long Term Extension Study of E2080 in Lennox-Gastaut Patients
NCT01405053	PHASE3	COMPLETED	Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
NCT04558580	PHASE3	WITHDRAWN	Safety Study of Rufinamide Given as an add-on Therapy to Treat Patients With Seizures
NCT02095899	PHASE2	WITHDRAWN	Effect of Rufinamide on Chronic Postthoracotomy Pain Syndrome
NCT02332174	PHASE1	COMPLETED	Pharmacokinetics and Tolerability of Rufinamide in Healthy Chinese Subjects
NCT06740825	PHASE1	COMPLETED	Study to Assess the Effect of a CYP3A Weak Inducer Rufinamide on Quizartinib Pharmacokinetics in Healthy Subjects
NCT03778424	Not specified	AVAILABLE	An Extended Access Program (EAP) for Participants Who Have Completed Rufinamide Study E2080-G000-303
NCT02175173	Not specified	COMPLETED	Post-marketing Surveillance of Long-term Administration of Inovelon Tablets in Patients With Lennox-Gastaut Syndrome
NCT03196466	Not specified	COMPLETED	Population Pharmacokinetics of Antiepileptic in Pediatrics

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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