Amifampridine - BioMarin

Drug Profile

Amifampridine - BioMarin

Alternative Names: 3,4-DAP; 3,4-DAP phosphate; 3,4-Diaminopyridine; Amifampridine phosphate; Firdapse; Pyridine-3,4-diamine-monophosphate; Zenas

Latest Information Update: 13 Jul 2018

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At a glance

  • Originator Assistance Publique Hopitaux de Paris
  • Developer BioMarin Pharmaceutical; Catalyst Pharmaceuticals; Fondazione Istituto Neurologico Carlo Besta
  • Class Aminopyridines; Small molecules
  • Mechanism of Action Acetylcholine stimulants; Potassium channel antagonists
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Myasthenia gravis; Lambert-Eaton myasthenic syndrome; Congenital myasthenic syndromes
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Lambert-Eaton myasthenic syndrome
  • Phase III Congenital myasthenic syndromes; Myasthenia gravis
  • Phase II Spinal muscular atrophy

Most Recent Events

  • 07 Aug 2018 Catalyst Pharmaceuticals expects to launch firdapse for Lambert-Eaton myasthenic syndrome in early 2019
  • 01 Jul 2018 Catalyst Pharmaceuticals initiates enrolment in a phase III trial for Myasthenia Gravis in USA (NCT03579966)
  • 24 Jun 2018 Biomarkers information updated
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