Quick answers
- What is Arimoclomol citrate used for?
- The FDA-approved label (Miplyffa, Acer Therapeutics Inc., March 18, 2026) says: “MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Arimoclomol citrate have a boxed warning?
- No. None of the 1 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Arimoclomol citrate?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Arimoclomol citrate been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Arimoclomol citrate?
- 1 company lists prescription products of Arimoclomol citrate in the FDA NDC Directory, including Acer Therapeutics Inc..
What Arimoclomol citrate is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
— FDA label: Miplyffa, Acer Therapeutics Inc., effective March 18, 2026. Read the full label on DailyMed →
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
Forms and how it is given
| Dosage forms | Capsule |
|---|---|
| Route | Oral |
| Prescription products | 4 in the FDA NDC Directory |
| Brand names | Miplyffa |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- Acer Therapeutics Inc.4 products
FDA approval history
- NDA: New Drug Application: how a new (usually brand-name) medicine is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| NDA214927 MIPLYFFA | Zevra Denmark | September 20, 2024 | — |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Miplyffa, Acer Therapeutics Inc., effective March 18, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →