Amifampridine

Summary

Amifampridine (CHEMBL354077) is an approved small molecule (ATC N07XX05); indicated across 4 conditions including myasthenia gravis and lambert-eaton myasthenic syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N07XX05
• Indications: 4 conditions
• Clinical trials: 5
• Chemistry: 109.13 Da · C5H7N3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL354077
Name	Amifampridine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	5918
ATC	N07XX05
Molecular formula	C5H7N3
Molecular weight	109.13
InChIKey	OYTKINVCDFNREN-UHFFFAOYSA-N

SMILES: C1=CN=CC(=C1N)N

IUPAC name: pyridine-3,4-diamine

Also known as: Amifampridina, Amifampridine, DAP, Dynamine, NSC-521760, Ruzurgi, SID51088028, SID56323425, SID50112814, AMIFAMPRIDINE, SID170466846, SID144206108

Parent form; salt/anhydrous children: CHEMBL3301611

Patent coverage: 3,287 distinct patent families (8,476 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 7,571 (89%) 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: 1 (assay-derived). Sample: Beta-lactamase.

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
P00811	6.15	Potency	707.9	nM	CHEMBL_ACT_4669502

Target pathways

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

Indications & clinical

Indications

4 indications (2 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
myasthenia gravis	4	MONDO:0009688	EFO:0004991
Lambert-Eaton myasthenic syndrome	4	MONDO:0018556	EFO:0020094
multiple sclerosis	3	MONDO:0005301	MONDO:0005301
botulism	2	MONDO:0005498	EFO:0005542

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE2	2
EARLY_PHASE1	1
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01511978	PHASE2	COMPLETED	Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
NCT05123053	PHASE2	COMPLETED	Firdapse for Post-BOTOX Vocal Weakness
NCT05769478	PHASE1	COMPLETED	Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA
NCT03937687	EARLY_PHASE1	COMPLETED	The Effects of a Combination of TeaCrine®, Dynamine, and Caffeine in Law Enforcement
NCT06048640	Not specified	COMPLETED	Effects of Dynamine Ingestion on Various Indices of Sustained Energy

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).