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Amifampridine - BioMarin

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Drug Profile

Amifampridine - BioMarin

Alternative Names: 3,4-DAP; 3,4-DAP phosphate; 3,4-DAPP; 3,4-Diaminopyridine; 3,4-Pyridinediamine-phosphate; Amifampridine phosphate; DAPP; DYD-301; Firdapse; Pyridine-3,4-diamine-monophosphate; Zenas

Latest Information Update: 22 Jul 2026

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At a glance

  • Originator Assistance Publique Hopitaux de Paris
  • Developer BioMarin Pharmaceutical; Catalyst Pharmaceuticals; DyDo Pharma; Fondazione Istituto Neurologico Carlo Besta; KYE Pharmaceuticals
  • Class Aminopyridines; Antineoplastics; 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 - Lambert-Eaton myasthenic syndrome; Myasthenia gravis; Congenital myasthenic syndromes
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 16 Jul 2026 Catalyst Pharmaceuticals has been acquired by Angelini Pharmaceuticals
  • 08 May 2026 Catalyst Pharmaceuticals and SERB settled patent litigation with Hetero Labs and Annora Pharma for amifampridine about approval in generic version
  • 07 May 2026 Catalyst Pharmaceuticals plans a clinical trial in May 2026

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