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: 05 Dec 2017

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At a glance

  • Originator Assistance Publique Hopitaux de Paris
  • Developer BioMarin Pharmaceutical; Catalyst Pharmaceutical; 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
  • Preregistration Congenital myasthenic syndromes
  • Phase II/III Myasthenia gravis
  • Phase II Spinal muscular atrophy

Most Recent Events

  • 27 Nov 2017 Efficacy data from a phase III trial in Lambert-Eaton myasthenic syndrome released by Catalyst Pharmaceuticals
  • 21 Nov 2017 Phase-II clinical trials in Spinal muscular atrophy in Italy (PO)
  • 08 Nov 2017 Catalyst Pharmaceuticals plans to initiate a phase III trial for Myasthenia gravis in USA in Q1 2018 (NCT03304054)
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