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Arimoclomol - Zevra Therapeutics

Drug Profile

Arimoclomol - Zevra Therapeutics

Alternative Names: Arimoclomol citrate; BRX-345; MIPLYFFA; OR-01; OR-04

Latest Information Update: 15 Jul 2024

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At a glance

  • Originator Biorex
  • Developer Food and Drug Administration; LadRx Corporation; Orphazyme; University of Kansas Medical Center
  • Class Anti-inflammatories; Antineoplastics; Antiparkinsonians; Chlorinated hydrocarbons; Hydroxylamines; Oxides; Piperidines; Pyridines; Small molecules
  • Mechanism of Action Heat shock transcription factor stimulants
  • Orphan Drug Status

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

    Yes - Niemann-Pick disease type C; Amyotrophic lateral sclerosis; Inclusion body myositis
  • New Molecular Entity Yes

Highest Development Phases

  • Preregistration Niemann-Pick disease type C
  • Phase II Gaucher's disease
  • No development reported Glioblastoma; Parkinson's disease
  • Discontinued Amyotrophic lateral sclerosis; Inclusion body myositis

Most Recent Events

  • 09 Jul 2024 US FDA plans to convene a meeting with Genetic Metabolic Diseases Advisory Committee (GeMDAC) to review the New Drug Application (NDA) for Arimoclomol in Niemann-Pick disease type C (NPC) in August 2024
  • 28 Mar 2024 Zevra Therapeutics announces intention to submit regulatory applications in EU and other parts of the world
  • 28 Mar 2024 Zevra Therapeutics plans to launch Arimoclomol for Niemann-Pick disease type C in USA
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