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Arimoclomol - Zevra Therapeutics

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Drug Profile

Arimoclomol - Zevra Therapeutics

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

Latest Information Update: 24 Sep 2024

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At a glance

  • Originator Biorex
  • Developer Food and Drug Administration; LadRx Corporation; Orphazyme; University of Kansas Medical Center; Zevra Therapeutics
  • Class Anti-inflammatories; Antineoplastics; Antiparkinsonians; Chlorinated hydrocarbons; Hydroxylamines; Oxides; Piperidines; Pyridines; Small molecules
  • Mechanism of Action
  • 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

  • Registered Niemann-Pick disease type C
  • Phase II Gaucher's disease
  • Discontinued Amyotrophic lateral sclerosis; Glioblastoma; Inclusion body myositis; Parkinson's disease

Most Recent Events

  • 23 Sep 2024 Discontinued - Preclinical for Glioblastoma in USA (PO) prior to September 2024 (Zevra Therapeutics pipeline, September 2024) (LadRx Corporation pipeline September 2024)
  • 23 Sep 2024 Discontinued - Preclinical for Parkinson's disease in Denmark (PO) prior to September 2024 (Zevra Therapeutics pipeline, September 2024) (LadRx Corporation pipeline September 2024)
  • 20 Sep 2024 Registered for Niemann-Pick disease type C (Combination therapy, In adolescents, In adults, In children) in USA (PO) - First Global Approval

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