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Arimoclomol · Miplyffa

Small molecule · developed by Zevra Denmark

Approved FDA, Sep 2024

ZVRA Developer

1 Registered trials

What is Arimoclomol?

Arimoclomol is a small molecule used in combination with miglustat for treating neurological manifestations of Niemann-Pick disease type C in patients 2 years and older. Its mechanism of action in NPC is unknown.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Miplyffa
Drug type: Small molecule
Therapy area: Neurology
Developer: Zevra Denmark (ZVRA)
FDA status: Approved as Miplyffa (September 20, 2024), NDA214927 · FDA label
UNII: EUT3557RT5

✅ FDA-approved uses

Neurological manifestations of Niemann-Pick disease type C

From the indications section of the FDA label (version of Mar 18, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT02612129	Phase 2/3	Niemann-Pick Disease, Type C	Completed	ZVRA

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Niemann-Pick Disease, Type C

More from Zevra Denmark

Osanetant Phase 1/2

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the ZVRA page.

Follow ZVRA, the company behind Arimoclomol

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z