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Dalfampridine

drug
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Also known as AmpyraAmpyra extended releaseEL-970FampridinaFampridineFampridine accordFampyraNSC-15041SID11110683SID11113806SID26751980SID50104658SID8139900SID90341267SID26751983SID104171097SID26747141SID144207991SID124879040

Summary

Dalfampridine (CHEMBL284348) is an approved small-molecule potassium channel blocker (ATC N07XX07) targeting KCNJ13, TRPV2, and KCNA2; indicated across 20 conditions including multiple sclerosis and stroke disorder.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: N07XX07
  • Targets: 15 (KCNJ13, TRPV2, KCNA2…)
  • Indications: 20 conditions
  • Clinical trials: 40
  • Chemistry: 94.11 Da · C5H6N2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL284348
NameDalfampridine
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID1727
ChEBICHEBI:34385
ATCN07XX07
Molecular formulaC5H6N2
Molecular weight94.11
InChIKeyNUKYPUAOHBNCPY-UHFFFAOYSA-N

SMILES: C1=CN=CC=C1N

IUPAC name: pyridin-4-amine

ChEBI definition: An aromatic amine that is pyridine bearing a single amino substituent at position 4. An orphan drug in the US, it is used to improve walking in adults with multiple sclerosis.

Pharmacological roles (ChEBI): avicide, potassium channel blocker, orphan drug.

Also known as: Ampyra, Ampyra extended release, Dalfampridine, EL-970, Fampridina, Fampridine, Fampridine accord, Fampyra, NSC-15041, SID11110683, SID11113806, SID26751980

Patent coverage: 8,765 distinct patent families (25,690 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 25,568 (100%) 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

GeneTargetActionpAffinityCancer dependencyUniProt
KCNJ13Kir7.1Antagonist0.1%O60928
TRPV2TRPV2Inhibition1.4%Q9Y5S1
KCNA2Kv1.2Pore blocker3.20.7%P16389
KCNA4Kv1.41.90.7%P22459
KCNA5Kv1.5Pore blocker3.60.2%P22460
KCNA7Kv1.73.60.5%Q96RP8
KCNA10Kv1.82.815.2%Q16322
KCNB1Kv2.1Pore blocker3.30%Q14721
KCNB2Kv2.22.80%Q92953
KCNC1Kv3.14.51.7%P48547
KCNC2Kv3.24.60.6%Q96PR1
KCNC3Kv3.32.90.1%Q14003
KCND1Kv4.120.1%Q9NSA2
KCND2Kv4.22.30.3%Q9NZV8
KCND3Kv4.32.45.5%Q9UK17

Broader ChEMBL bioactivity targets: 12 (assay-derived). Sample: Microtubule-associated protein tau, Survival motor neuron protein, Prelamin-A/C, Thyrotropin receptor, Menin/Histone-lysine N-methyltransferase MLL, Muscarinic acetylcholine receptor M1, Potassium voltage-gated channel subfamily A member 1, Cytochrome P450 2D6, Cruzipain, Aldehyde dehydrogenase 1A1, Mitogen-activated protein kinase 1, Lethal factor.

Bioactivity

ChEMBL activities: 10 potent at pChembl ≥ 5 of 14 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
CYP2D67.2Potency63.1nMCHEMBL_ACT_4998352
CYP2D67.2AC5063.1nMCHEMBL_ACT_5987240
TSHR6.9Potency125.9nMCHEMBL_ACT_3918937
LMNA6.05Potency891.3nMCHEMBL_ACT_3640364
SMN16Potency1000nMCHEMBL_ACT_3877503
P257795.5Potency3162nMCHEMBL_ACT_4549909
P257795.45Potency3548nMCHEMBL_ACT_3978300
P159175.1Potency7943nMCHEMBL_ACT_4671085
MAPT5.05Potency8912nMCHEMBL_ACT_4513917
MAPK15Potency10000nMCHEMBL_ACT_4705401

Target pathways

Aggregated over 15 target gene(s): KCNJ13, TRPV2, KCNA2, KCNA4, KCNA5, KCNA7, KCNA10, KCNB1, KCNB2, KCNC1, KCNC2, KCNC3, KCND1, KCND2, KCND3.

Top Reactome pathways

12 total, by targets touching each:

PathwayTargetsGenes
Neuronal System13KCNA10, KCNA2, KCNA4, KCNA5, KCNA7, KCNB1, KCNB2, KCNC1, KCNC2, KCNC3, KCND1, KCND2, KCND3
Potassium Channels13KCNA10, KCNA2, KCNA4, KCNA5, KCNA7, KCNB1, KCNB2, KCNC1, KCNC2, KCNC3, KCND1, KCND2, KCND3
Voltage gated Potassium channels13KCNA10, KCNA2, KCNA4, KCNA5, KCNA7, KCNB1, KCNB2, KCNC1, KCNC2, KCNC3, KCND1, KCND2, KCND3
Muscle contraction4KCNA5, KCND1, KCND2, KCND3
Cardiac conduction4KCNA5, KCND1, KCND2, KCND3
Phase 1 - inactivation of fast Na+ channels3KCND1, KCND2, KCND3
Metabolism2KCNB1, KCNC2
Integration of energy metabolism2KCNB1, KCNC2
Glucagon-like Peptide-1 (GLP1) regulates insulin secretion2KCNB1, KCNC2
Regulation of insulin secretion2KCNB1, KCNC2
TRP channels1TRPV2
Phase 3 - rapid repolarisation1KCNA5

Dominant GO biological processes

GO termTargets
monoatomic ion transport15
monoatomic ion transmembrane transport15
potassium ion transport14
potassium ion transmembrane transport14
transmembrane transport14
action potential13
protein homooligomerization13
potassium ion export across plasma membrane4
regulation of presynaptic membrane potential4
optic nerve development3
protein tetramerization3
regulation of monoatomic ion transmembrane transport2
regulation of membrane potential2
neuronal action potential2
sensory perception of pain2

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

IndicationPhaseMONDOEFO
multiple sclerosis4MONDO:0005301MONDO:0005301

14 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)PhaseMONDOEFO
stroke disorder3MONDO:0005098EFO:0000712
relapsing-remitting multiple sclerosis3MONDO:0005314EFO:0003929
spinal cord injury3MONDO:0043797EFO:1001919
secondary progressive multiple sclerosis3MONDO:0000450EFO:0008522
obstructive sleep apnea syndrome2MONDO:0007147EFO:0003918
Guillain-Barre syndrome2MONDO:0016218EFO:0007292
nervous system injury2MONDO:0044745EFO:0009490
prostate disorder2MONDO:0003105EFO:0009602
injury2MONDO:0021178EFO:0000546
carpal tunnel syndrome2MONDO:0007275EFO:0004143
spinal muscular atrophy2MONDO:0001516MONDO:0019079
cerebral palsy1MONDO:0006497EFO:1000632
motor neuron disorder1MONDO:0020128EFO:0003782
kidney failure1MONDO:0001106HP:0000083

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

Clinical trials

Total trials: 40.

Phase distribution

PhaseTrials
PHASE215
Not specified8
PHASE2/PHASE36
PHASE45
PHASE32
PHASE12
PHASE1/PHASE21
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT01480076PHASE4COMPLETEDOpen-Label Study to Assess the Effect of Long-Term Prolonged-Release Fampridine (BIIB041) on Quality of Life as Reported by Participants With Multiple Sclerosis
NCT01975324PHASE4COMPLETEDA New Medicine to Treat Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)
NCT02208050PHASE4COMPLETEDA Study of the Effectiveness of Fampridine in Improving Upper Limb Function in MS
NCT02259361PHASE4UNKNOWNEfficacy of Dalfampridine on Upper Extremity Function in Patients With MS
NCT02849782PHASE4COMPLETEDShort and Long Term Multiple Outcomes in Persons With Multiple Sclerosis Treated by Fampridine.
NCT03701581PHASE2/PHASE3RECRUITING4-aminopyridine Treatment for Nerve Injury
NCT01337986PHASE2/PHASE3COMPLETEDAmpyra for Optic Neuritis in Multiple Sclerosis
NCT01645787PHASE2/PHASE3COMPLETEDShort and Long Term Treatment With 4-AP in Ambulatory SMA Patients
NCT02006160PHASE2/PHASE3COMPLETEDEffects of Dalfampridine on Cognition in Multiple Sclerosis
NCT02219932PHASE3COMPLETEDEfficacy and Safety Study of Prolonged-Release Fampridine in Participants With Multiple Sclerosis
NCT03899584PHASE3UNKNOWNHigh Doses of 4-aminopyridine in Clinically Complete Chronic Spinal Cord Injury Patients.
NCT04026568PHASE2/PHASE3TERMINATEDA Single Dose Pharmaco-Diagnostic for Peripheral Nerve Continuity After Trauma
NCT05859802PHASE2/PHASE3COMPLETEDEffects of Physical Therapy and Dalfampridine on Functional Mobility in Non Ambulatory Persons With Multiple Sclerosis
NCT06003166PHASE2RECRUITING4-AP Peripheral Nerve Crossover Trial
NCT06294821PHASE2NOT_YET_RECRUITING4AP for Carpal Tunnel Syndrome (CTS)
NCT06333171PHASE2RECRUITING4-aminopyridine for Skin Wound Healing
NCT06596434PHASE2RECRUITING4-Aminopyridine to Treat Skin Burns
NCT06853015PHASE1/PHASE2RECRUITINGDouble Dose 4-AP on Functional Recovery After Spinal Cord Injury
NCT00056810PHASE2COMPLETEDAssessment of Chronic Guillain-Barre Syndrome Improvement With Use of 4-aminopyridine
NCT01444300PHASE2COMPLETEDDalfampridine for Imbalance in Multiple Sclerosis
NCT01543750PHASE2WITHDRAWN4-Aminopyridine in Episodic Ataxia Type 2
NCT01576354PHASE2UNKNOWNCharacterization of the Effects of Prolonged-release Fampridine on Ambulatory Function in Patients With Multiple Sclerosis
NCT01621113PHASE2COMPLETEDCombination Therapy With Dalfampridine and Locomotor Training for Chronic, Motor Incomplete Spinal Cord Injury
NCT02166346PHASE2COMPLETEDSafety and Efficacy of Sustained Release Dalfampridine in Transverse Myelitis (Re-Launch)
NCT02280096PHASE2COMPLETEDEfficacy and Safety of 4-aminopyridine on Cognitive Performance and Motor Function of Patients With Multiple Sclerosis
NCT02391961PHASE2COMPLETEDStudy and Treatment of Visual Dysfunction and Motor Fatigue in Multiple Sclerosis
NCT02656160PHASE2COMPLETEDEffect of Dalfampridine (4-AP) on Genioglossus Muscle Activity in Healthy Adults
NCT03578354PHASE2WITHDRAWN4-Aminopyridine, Atenolol, or Placebo in Patients With Vestibular Migraine
NCT03658408PHASE2WITHDRAWN4-aminopyridine Treatment for Nerve Injury From Radical Retro-Pubic Prostatectomy
NCT02868567PHASE1ACTIVE_NOT_RECRUITINGUse of Dalfampridine in Primary Lateral Sclerosis
NCT07171203PHASE1RECRUITINGPreoperative Imatinib and Fampridine in KIT Mutant Gastrointestinal Stromal Tumor
NCT05447676EARLY_PHASE1UNKNOWNEffects of 4-AP on Functional SCI Recovery
NCT07532460Not specifiedNOT_YET_RECRUITINGThe Role of Potassium Channels in Working Memory Impairments of Chronic Cocaine Users
NCT01480063Not specifiedCOMPLETEDAn Observational Study to Collect Information on Safety and to Document the Drug Utilization of Fampyra (BIIB041) When Used In Routine Medical Practice
NCT01532154Not specifiedTERMINATEDFampridine Pregnancy Exposure Registry
NCT01720849Not specifiedUNKNOWNEfficiency of 4-aminopyridin (Fampyra) on Gait, Vision, Cognition, Fatigue and Micturation in Patients With Multiple Sclerosis
NCT01811706Not specifiedCOMPLETEDDalfampridine and Gait in Spinocerebellar Ataxias
NCT03164018Not specifiedCOMPLETEDFampridine in MS Patients: A Cognition, Fatigue, Depression and Quality of Life Analysis
NCT03847545Not specifiedCOMPLETEDMuscle Strain in Multiple Sclerosis Patients Measured by Ultrasound Speckle Tracking Technique
NCT05613114Not specifiedCOMPLETEDEffect of Dalfampridine in Patients With Hereditary Spastic Paraplegia

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.

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

50 molecules share ≥1 primary target. Top 50 by shared-target count:

MoleculeSourceStatusShared targets
FLECAINIDEChEMBL + PubChemPhase 4 (approved)KCNA5, KCNA7
QUINIDINEChEMBL + PubChemPhase 4 (approved)KCNA5, KCNA7
VERNAKALANTChEMBLPhase 4 (approved)KCNA5, KCND2
BelzutifanPubChemApprovedKCNA5, KCND3
DARIFENACINChEMBL + PubChemPhase 4 (approved)KCND3
DARUNAVIRChEMBL + PubChemPhase 4 (approved)KCND3
DEFERASIROXChEMBL + PubChemPhase 4 (approved)KCND3
DronedaroneChEMBL + PubChemPhase 4 (approved)KCNA5
DULOXETINEChEMBL + PubChemPhase 4 (approved)KCND3
NEBIVOLOLChEMBL + PubChemPhase 4 (approved)KCND3
PALIPERIDONEChEMBL + PubChemPhase 4 (approved)KCND3
SOLIFENACINChEMBL + PubChemPhase 4 (approved)KCND3
SUNITINIBChEMBL + PubChemPhase 4 (approved)KCND3
TOLTERODINEChEMBL + PubChemPhase 4 (approved)KCND3
VARDENAFILChEMBL + PubChemPhase 4 (approved)KCND3
NIFEDIPINEChEMBLPhase 4 (approved)KCNA5
SERTINDOLEChEMBLPhase 4 (approved)KCNA5
BERGAPTENChEMBLPhase 3KCNA2
CANNABINOLChEMBLPhase 3TRPV2
BMS-919373ChEMBLPhase 2KCNA5
CANNABIDIVARINChEMBLPhase 2TRPV2
CANNABIGEROLChEMBLPhase 2TRPV2
TETRAHYDROCANNABIVARINChEMBLPhase 2TRPV2
AlfuzosinPubChemApprovedKCND3
AlvimopanPubChemApprovedKCND3
AmbrisentanPubChemApprovedKCND3
AmiodaronePubChemApprovedKCNA7
CannabidiolPubChemApprovedTRPV2
DesvenlafaxinePubChemApprovedKCND3
DiltiazemPubChemApprovedKCNA5
DofetilidePubChemApprovedKCND3
EtravirinePubChemApprovedKCND3
EverolimusPubChemApprovedKCND3
LacosamidePubChemApprovedKCND3
LamotriginePubChemApprovedKCND3
LapatinibPubChemApprovedKCND3
LidocainePubChemApprovedKCNA5
MoxifloxacinPubChemApprovedKCND3
NelfinavirPubChemApprovedKCND3
NilotinibPubChemApprovedKCND3
PalonosetronPubChemApprovedKCND3
PropafenonePubChemApprovedKCND3
QuininePubChemApprovedKCNB2
RaltegravirPubChemApprovedKCND3
SildenafilPubChemApprovedKCND3
SilodosinPubChemApprovedKCND3
SitagliptinPubChemApprovedKCND3
TadalafilPubChemApprovedKCND3
VerapamilPubChemApprovedKCNA7
ZuranolonePubChemApprovedKCND3

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot