Arimoclomol

NERVOUS SYSTEM

Key facts

Arimoclomol is an onp drug catalogued in DrugCentral. It was first approved by the FDA in 2024. Its mechanism of action is classified as Organic Cation Transporter 2 Inhibitors. Recorded indications include Niemann-Pick disease, type C.

• Approval status: onp (first approved 2024 by the FDA).
• Mechanism of action: Organic Cation Transporter 2 Inhibitors.
• Indications: Niemann-Pick disease, type C.
• ATC code: N07XX17 (Other nervous system drugs).
• Molecular formula: C14H20ClN3O3; molecular weight 313.79 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Niemann-Pick disease, type C

Mechanism & targets

• Mechanism of action (MoA): Organic Cation Transporter 2 Inhibitors

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2024-09-20	FDA	—	Zevra Therapeutics Inc.	Yes

ATC classification

ATC code	Therapeutic hierarchy
N07XX17	NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label
• FDA label: NPC1, NPC2

Chemical structure

• Molecular formula: C₁₄H₂₀ClN₃O₃
• Molecular weight: 313.79 g/mol
• CAS number: —
• InChIKey: SGEIEGAXKLMUIZ-XUVDNFPMSA-N
• PubChem CID: 208924

O[C@@H](CO\N=C(/Cl)C1=C[N+]([O-])=CC=C1)CN1CCCCC1

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Arimoclomol used for?

DrugCentral lists the following indications for Arimoclomol: Niemann-Pick disease, type C.

How does Arimoclomol work?

Arimoclomol is classified as Organic Cation Transporter 2 Inhibitors.

When was Arimoclomol first approved?

Arimoclomol was first approved by the FDA in 2024.

What is the chemical formula of Arimoclomol?

Arimoclomol has the molecular formula C14H20ClN3O3 and a molecular weight of 313.79 g/mol.

Sources

• DrugCentral drug card 5857 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL2107726 — compound record and bioactivity
• PubChem CID 208924 — chemical identifiers
• DrugBank DB05025 — drug record
• FDA UNII EUT3557RT5 — substance identity
• DailyMed: FDA labels for Arimoclomol — current prescribing information (label search)
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.