Uses
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: US FDA prescribing information
- Presentations
- 4
- Brands
- 1
- Manufacturers
- 1
- Available from stock
- 0
Arimoclomol is a prescription medicine indicated 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. MedsAcross supplies Arimoclomol for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.
Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review
Key facts
- Pharmacological class
- Organic Cation Transporter 2 Inhibitors
- ATC classification
- N07XX17 Arimoclomol · Other nervous system medicines, subgroup N07X, subgroup N07XX
- US FDA first approval
- 20 September 2024
- EU status
- Opinion
- Orphan designation
- Yes
- WHO essential medicine
- Not recorded
- India (CDSCO)
- Not yet confirmed against CDSCO approval lists
- Routes
- Oral
- Dosage forms
- Capsule
- Identifiers
- UNII EUT3557RT5CAS 289893-25-0DrugBank DB05025Q4790694
Clinical information
From the regulator-approved product information. Each section names its source.
How it works
The mechanism(s) by which arimoclomol exerts its clinical effects in patients with NPC is unknown.
Source: US FDA prescribing information
Dosing overview
Recommended MIPLYFFA oral dosage, in combination with miglustat, for patients with actual body weight of: 8 kg to 15 kg, is 47 mg three times a day > 15 kg to 30 kg, is 62 mg three times a day > 30 kg to 55 kg, is 93 mg three times a day > 55 kg, is 124 mg three times a day Administer with or without food. See full prescribing information for recommended dosage in patients with an eGFR ≥ 15 to < 50 mL/minute. See full prescribing information for instructions on preparation and administration.
Source: US FDA prescribing information
Contraindications
None. None.
Source: US FDA prescribing information
Warnings and precautions
Hypersensitivity Reactions: Urticaria and angioedema have been reported. Discontinue MIPLYFFA in patients who develop these adverse reactions. Embryofetal Toxicity: May cause fetal harm. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention. Increased Creatinine without Affecting Glomerular Function: Mean increases in serum creatinine of 10-20% have been reported. Use alternative measures to assess renal function which are not based on creatinine.
Source: US FDA prescribing information
Side effects
The following clinically significant adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions. Increased Creatinine without Affecting Glomerular Function. Most common adverse reactions (≥15%) are: Upper respiratory tract infection, diarrhea, and decreased weight.
Source: US FDA prescribing information
Interactions
Substrates of the Organic Cationic Transporter 2 (OCT2 substrates): Monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Source: US FDA prescribing information
Storage
Store MIPLYFFA at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F). Store in original container. Protect from light.
Source: US FDA prescribing information
For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.
All presentations
4 presentations of Arimoclomol, sorted by availability.
| Medicine | Manufacturer | Register | Availability |
|---|---|---|---|
| Miplyffa 124 mg capsuleOral | Acer Therapeutics | US FDA | Available on request |
| Miplyffa 47 mg capsuleOral | Acer Therapeutics | US FDA | Available on request |
| Miplyffa 62 mg capsuleOral | Acer Therapeutics | US FDA | Available on request |
| Miplyffa 93 mg capsuleOral | Acer Therapeutics | US FDA | Available on request |
Brands by market
1 brand names across 1 markets.
United States
Manufacturers
Regulatory status
Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.
| Register | Status | Source |
|---|---|---|
| US FDA | First approved 20 September 2024 | Drugs@FDA |
| EU (EMA) | Opinion | EMA medicines data |
| India (CDSCO) | Not yet confirmed against CDSCO approval lists | — |
| United States | Registered | US FDA (NDC directory, openFDA)Checked 12 November 2026 |
Questions and answers
Is Arimoclomol available for export from India?
Yes, on request. MedsAcross lists 4 presentations of Arimoclomol and sources them for licensed hospitals, importers and physicians against valid import documents.
What brand names does Arimoclomol have?
The catalogue lists 1 brand names for Arimoclomol, including Miplyffa.
Is Arimoclomol approved by the US FDA?
Yes. The US FDA first approved Arimoclomol on 20 September 2024, according to Drugs@FDA.
Does Arimoclomol need cold chain shipping?
No listed presentation of Arimoclomol is recorded as needing a cold chain. Storage conditions are shown on each presentation page.
Which ATC class is Arimoclomol in?
Arimoclomol is classified under ATC code N07XX17 (Arimoclomol).
References
- MIPLYFFA prescribing information (Acer Therapeutics). U.S. National Library of Medicine, DailyMed. Retrieved 18 March 2026
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