Arimoclomol: uses, dosing, side effects & brands
Arimoclomol is a medicine sold in the U.S. under one brand, for neurodegenerative diseases and type c niemann-pick disease. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
Key facts
- Treats
- Neurodegenerative Diseases and Type C Niemann-Pick Disease
- Available as
- Capsule
- Sold as
- Miplyffa
- Prescription?
- Prescription only
- Generic available?
- Not in our catalog
How arimoclomol is dosed
From the FDA label for Miplyffa (application NDA214927). Other arimoclomol products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
Recommended MIPLYFFA oral dosage, in combination with miglustat, for patients with actual body weight of ( 2.1 ): 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. ( 2.1 ) See full prescribing information for recommended dosage in patients with an eGFR ≥ 15 to < 50 mL/minute. ( 2.2 ) See full prescribing information for instructions on preparation and administration. ( 2.3 ) 2.1 Recommended Dosage The recommended oral dosage of MIPLYFFA, in combination with miglustat, for patients with an 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 MIPLYFFA with or without food. Missed Dose If a dose of MIPLYFFA is missed, advise the patient to skip the missed dose and to resume taking the prescribed dose at the next scheduled time. 2.2 Recommended Dosage in Patients with Renal Impairment The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥ 50 mL/minute is the same as the recommended dosage in patients with normal renal function [see Dosage and Administration ( 2.1 )]. For patients with an eGFR ≥ 15 to < 50 mL/minute, the recommended oral dosage of…
Arimoclomol side effects
The following clinically significant adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions [see Warning and Precautions ( 5.1 )]. Increased Creatinine without Affecting Glomerular Function [see Warnings and Precautions ( 5.3 )]. Most common adverse reactions (≥15%) are: Upper respiratory tract infection, diarrhea, and decreased weight. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of MIPLYFFA was evaluated in a randomized, double-blind, placebo-controlled, 12-month trial (Trial 1), which included 50 patients 2 to 19 years old with NPC [see Clinical Studies ( 14 )] . Patients received weight-adjusted doses of MIPLYFFA (31 to 124 mg orally three times daily); 28 of the patients were exposed to MIPLYFFA for one year. In Trial 1, 78% of patients received miglustat. Forty-one out of 50 patients that were enrolled in Trial 1 continued into an open-label extension trial, which included 39 patients treated with MIPLYFFA for…
Who shouldn’t take arimoclomol
None. None. ( 4 )
Arimoclomol drug interactions
Substrates of the Organic Cationic Transporter 2 (OCT2 substrates) : Monitor for adverse reactions and reduce the dosage of the OCT2 substrate. ( 7.1 ) 7.1 Effect of MIPLYFFA on Other Drugs Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates [see Clinical Pharmacology ( 12.3 )] . When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Every arimoclomol product we track (1)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
Only one: Miplyffa.
What arimoclomol pills look like
Imprint codes, colour and shape from the FDA’s labelling data. Match the imprint on your pill — or search any imprint.
What people report to the FDA about arimoclomol
The FDA Adverse Event Reporting System (FAERS) collects reports from patients and clinicians. It holds 11 reports naming arimoclomol, and the FDA flagged 100% of those reports as serious. The effects reported most often — leaving out reports about overdose, misuse or the condition being treated, which dominate the raw list for common medicines:
- pneumonia2 reports
- sepsis2 reports
- vomiting2 reports
- abdominal abscess1 reports
- acute kidney injury1 reports
- acute respiratory failure1 reports
- anaemia1 reports
- cholelithiasis1 reports
Read these as a signal, not a rate. A report does not mean arimoclomol caused the effect — anyone can file one, and many describe people taking several medicines for several conditions. Crucially there is no denominator: FAERS does not record how many people took the drug, so these counts cannot be turned into “X% of patients” — a bigger number often just means a more widely used or more talked-about drug. Duplicates exist, and publicity drives reporting. For what is actually established, read the FDA label section above.
Source: openFDA drug/event (FAERS), retrieved July 25, 2026.
Related calculators
Frequently asked questions
What is arimoclomol?
MIPLYFFA capsules contain arimoclomol citrate. Arimoclomol citrate is a crystalline powder of white to off-white color that is freely soluble in water.
Can you take arimoclomol with other medicines?
It depends on the medicine. We check it against the FDA labels rather than guessing: our interaction checker searches each drug's own label for the other and quotes what it says, naming the section it came from. Run arimoclomol against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What forms does arimoclomol come in?
Across the brands we track, arimoclomol is currently marketed as capsule, per the FDA's National Drug Code Directory. Each form is dosed differently — follow the label for the exact product you were prescribed.
Is there a generic arimoclomol?
We do not currently list a generic-labelled arimoclomol product. That does not always mean none exists — it means none appears under a generic name in the FDA data we track. Ask your pharmacist.
Cite this page
- APA
- pharmaranks. (2026, July 24). Arimoclomol: uses, dosing, side effects & brands. https://pharmaranks.com/drugs/arimoclomol
- MLA
- “Arimoclomol: uses, dosing, side effects & brands.” pharmaranks, 24 July 2026, https://pharmaranks.com/drugs/arimoclomol.
We summarise public FDA and NIH sources — for a clinical claim, cite the primary source we link to as well.
Sources: FDA openFDA drug label, National Drug Code Directory, and Enforcement (recall) database. This page reproduces public FDA data and is not medical advice. Dosing is set by your prescriber.
Read the full FDA label for arimoclomol on DailyMed (NIH) ↗.