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PlainMeds

Prescription medicine

Arimoclomol Brand names: Miplyffa

Arimoclomol (Miplyffa) is a prescription medicine with an FDA approval on record since 2024. No generic is listed in FDA's NDC Directory.

  • No generic listed

Check it at the source: the arimoclomol FDA label on DailyMed (effective March 18, 2026, Acer Therapeutics Inc.); the NDA 214927 record on Drugs@FDA; every arimoclomol label on DailyMed.

Highlights of the record

FDA label of March 18, 2026; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026

Used for

From the label: indications and usage

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.

Forms and strengths

  • 47 mg
  • 62 mg
  • 93 mg
  • 124 mg

Capsule.

On the market

Earliest approval on file
September 20, 2024 Miplyffa, Zevra Denmark; Drugs@FDA’s file, where older or withdrawn approvals can be missing
Generic approvals
No generic approved
Labelers listed (NDC)
1 of which 0 under generic applications, 1 under brand applications, repackagers included
Last listed patent
November 19, 2041 Miplyffa capsules; not a generic launch date

Supply and safety

Pharmacy cost (NADAC)
Not in the survey
Shortage (FDA)
No current entry
Recalls by its makers (FDA)
No recall recorded
FAERS reports
45 through July 30, 2026

What is arimoclomol used for?

From the label: indications and usage

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.

Source: FDA drug label (MIPLYFFA), NDA214927 (Zevra Denmark), as published by Acer Therapeutics Inc., effective March 18, 2026 (SPL set 5feffc0e-453d-47fa-91dd-38d4952309bc), via openFDA; the full label on DailyMed

Is there a generic for arimoclomol (Miplyffa)?

No generic arimoclomol is approved (Drugs@FDA, October 8, 2026).

The Orange Book lists 4 patents for Miplyffa capsules: the compound patent runs to November 19, 2041. A patent or exclusivity date is not the date a generic goes on sale: court cases and settlements move it either way.

  • 2024 earliest approval on file
  • 2041 last listed patent

Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)

What do FAERS reports show for arimoclomol?

The FDA Adverse Event Reporting System holds 45 reports that list arimoclomol among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 3 are coded as a death and 10 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.

The most reported reactions

  • Diarrhoea (named in the label)6
  • Vomiting5
  • Product Size Issue4
  • Insomnia3
  • Pneumonia Aspiration3
  • Death (an outcome recorded in the report)2
  • Fall2
  • Fatigue2

Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026

What next?

Arimoclomol side effects
the label's list beside FAERS reports
Check an interaction
what two or more labels say about each other