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amifampridine phosphate

Trade name: firdapse

Small moleculeapprovedOrphan Drug FDABreakthrough Therapy FDA

Approved

Nov 28, 2018

Amifampridine (Firdapse), currently approved in the European Union, is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, a rare autoimmune disease with the primary symptoms of muscle weakness. In LEMS, the body’s own immune system attacks connections between nerves and muscles and disrupts the ability of nerve cells to send signals to muscle cells. Amifampridine blocks voltage-dependent potassium channels, thereby prolonging pre-synaptic cell membrane depolarization. Prolonging the action potential enhances the transport of calcium into the nerve ending. The resulting increase in intracellular calcium concentrations facilitates exocytosis of acetylcholine containing vesicles, which in turn enhances neuromuscular transmission. Amifampridine phosphate has been granted Orphan Drug Designation and Breakthrough Therapy designation by the FDA for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). — NCATS

Clinical trial activity

28 trials · 27 clinical orgs · 2 marketing orgs

Phase 1
1
Phase 2
14
Phase 3
10
Phase 4
0

Earliest trial started Aug 1, 1994 (NCT00004832)

Timeline

1990s

  1. Dec 18, 1990

    Orphan Drug Designation

  2. Jan 1, 1996

    Earliest Phase 3 Sponsor(trial)

2000s

  1. Jan 1, 2006

    Earliest Phase 2 Sponsor(trial)

2010s

  1. Nov 28, 2018

    Catalyst Pharmaceuticals, Inc. — Earliest FDA Approval

  2. Nov 28, 2018

    Catalyst Pharmaceuticals, Inc. — NDA Secondary Org

  3. Nov 28, 2018

    Catalyst Pharmaceuticals, Inc. — NDA Organization

  4. May 6, 2019

    Jacobus Pharmaceutical — Marketing Organization

2020s

  1. Feb 1, 2022

    Jacobus Pharmaceutical — NDA Secondary Org

  2. Jan 1, 2023

    Earliest Phase 1 Sponsor(trial)

Indications

Approved for

Studied for

Mechanism of action

Approval history

  • approvedPriority reviewBreakthroughNov 28, 2018

Chemistry & pharmacology

Loading structure…

SMILES

Nc1ccncc1N
Mol. weight
109.13 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Achiral Molecule
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

Sources

Also known as

  • 1-(diethylaminopropyl)-4-phenylpiperazine
  • 3,4-dap
  • 3,4-dapp
  • 3,4-dapp
  • 3,4 diaminopyridine
  • 3,4 diaminopyridine
  • 3,4-diaminopyridine
  • 3,4-diaminopyridine
  • 3,4-diaminopyridine
  • 3,4-diammoniopyridinium
  • 3,4-diammoniopyridinium
  • 4-dap
  • 4-dap
  • 4-diaminopyridine
  • amifampridin
  • amifampridine
  • amifampridine
  • amifampridine
  • amifampridine
  • amifampridine
  • amifampridine
  • amifampridine
  • amifampridine phosphate
  • amifampridine phosphate
  • amifampridine phosphate
  • amifampridine phosphate
  • amifampridine phosphate
  • amifampridine phosphate
  • dap
  • dap
  • dap
  • dapp
  • dapp
  • dapp
  • diaminopyridine
  • diaminopyridine
  • firdapse
  • firdapse
  • pyridine-3,4-diamine
  • pyridine-3,4-diamine

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT05769478Phase 1Sep 15, 2023Wake Forest University
NCT05919407Phase 3Mar 22, 2023Leiden University, NMD Pharma A/S
NCT05123053Phase 2Oct 28, 2021Augusta University
NCT03819660Phase 2Mar 7, 2019Carlo Besta Neurological Institute, Catalyst Pharmaceuticals, Inc.
NCT03781479Phase 2Jan 21, 2019Carlo Besta Neurological Institute, Catalyst Pharmaceuticals, Inc.
NCT03579966Phase 3Jul 1, 2018Carlo Besta Neurological Institute, Catalyst Pharmaceuticals, Inc.
NCT03304054Phase 3Mar 7, 2018Carlo Besta Neurological Institute, Catalyst Pharmaceuticals, Inc.
NCT03062631N/AFeb 21, 2017Jacobus Pharmaceutical, University of California, Davis
NCT02970162Phase 3Nov 1, 2016Catalyst Pharmaceuticals, Inc., University of California, Los Angeles
NCT02562066Phase 3Jan 1, 2016Catalyst Pharmaceuticals, Inc., Emory University, Johns Hopkins University
NCT02189720N/AJul 5, 2014Catalyst Pharmaceuticals, Inc., University of California, Los Angeles
NCT02012933N/ADec 11, 2013Jacobus Pharmaceutical, Oregon Health and Science University
NCT01765140N/AJan 6, 2013Duke University
NCT01511978Phase 2Jan 1, 2012Jacobus Pharmaceutical
NCT01441557Phase 2/Phase 3 (Phase 3)Sep 1, 2011University of Picardie Jules Verne
NCT01377922Phase 3Jun 1, 2011Catalyst Pharmaceuticals, Inc.
NCT00994916N/AOct 8, 2009Jacobus Pharmaceutical, University of Pittsburgh
NCT00701077Phase 3Jul 1, 2008University of Paris
NCT00342381Phase 2Jun 1, 2006Aarhus University, The Danish Multiple Sclerosis Society
NCT01378546N/AMay 1, 2005Columbia University
Showing 20 of 28 trials
Page 1 / 2

Organizations

Research & Development (27)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Catalyst Pharmaceuticals, Inc.For profit8832011
Jacobus PharmaceuticalFor profit6122000
Carlo Besta Neurological InstituteAcademic/Hospital4022018
Duke UniversityAcademic/Hospital2111994
University of California, DavisAcademic/Hospital2212000
University of California, Los AngelesAcademic/Hospital2022014
University of ParisAcademic/Hospital2212005
Aarhus UniversityAcademic/Hospital1112006
Augusta UniversityAcademic/Hospital1112021
CHU Henri MondorAcademic/Hospital1012005
Columbia UniversityAcademic/Hospital1112005
Dartmouth UniversityAcademic/Hospital1112004
Emory UniversityAcademic/Hospital1012016
Food and Drug AdministrationGovernment1111994
Johns Hopkins UniversityAcademic/Hospital1012016
Leiden UniversityAcademic/Hospital1112023
Mayo ClinicAcademic/Hospital1011996
National Center for Research Resources (NCRR)Government1111996
NMD Pharma A/SFor profit1012023
Oregon Health and Science UniversityAcademic/Hospital1112013
27 organizations
Page 1 / 2

Marketing (4)

OrganizationOrg typeRelationshipDate
Catalyst Pharmaceuticals, Inc.For profitNDA2Nov 28, 2018
Catalyst Pharmaceuticals, Inc.For profitNDANov 28, 2018
Jacobus PharmaceuticalFor profitMKTGMay 6, 2019
Jacobus PharmaceuticalFor profitNDA2Feb 1, 2022