amifampridine phosphate (firdapse) Report issue

Small molecule Orphan Drug FDA Approved FDA Priority Review FDA
AACT ChEMBL Drug Central Drugs@FDA KEGG MeSH PubChem

Active Ingredient History

NOW
  • Now
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

Chemistry

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Pharmacology

Drug Pricing (per unit)

United States

$56.8892 - $127.5129
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Alternative names

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

Organizations (27)

Research & Development (27)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (2)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (13)

Approved Indications (1)


 

Lambert-Eaton Myasthenic Syndrome (approved 2009)

Experimental Indications - Clinical Trial Phases 1-4 (13)


 

Botulinum Toxins, Type A (Phase 2)

Botulism (Phase 2/Phase 3)

Brugada Syndrome (Phase 3)

Fatigue (Phase 2)

Lambert-Eaton Myasthenic Syndrome (Phase 3)

Multiple Sclerosis (Phase 3)

Muscular Atrophy (Phase 2)

Muscular Atrophy, Spinal (Phase 2)

Myasthenia Gravis (Phase 3)

Myasthenic Syndromes, Congenital (Phase 3)

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating (Phase 3)

Spinal Muscular Atrophies of Childhood (Phase 2)

Vocal Cord Dysfunction (Phase 2)

Overview

  • Highest Phase: Phase 3
  • # of Trials: 28
  • # of Trial Organizations: 27

Trials by Phase

Trial Phase Start Date Organizations Indications

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