Drug Profile
Arimoclomol - Zevra Therapeutics
Alternative Names: Arimoclomol citrate; BRX-345; MIPLYFFA; OR-01; OR-04Latest Information Update: 06 Mar 2024
Price :
$50
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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
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Preregistration Niemann-Pick disease type C
- Phase II Gaucher's disease
- Preclinical Parkinson's disease
- No development reported Glioblastoma
- Discontinued Amyotrophic lateral sclerosis; Inclusion body myositis
Most Recent Events
- 04 Mar 2024 FDA assigns PDUFA action date of (21/09/2024) for arimoclomol for Niemann-Pick disease type C
- 04 Mar 2024 The US FDA extends the review period for the NDA for arimoclomol for Niemann-Pick disease type C
- 08 Jan 2024 The US FDA accepts re-summited NDA for arimoclomol for Niemann-Pick disease type C for review