Arimoclomol
Arimoclomol is a prescription capsule medicine, taken together with miglustat, to treat the neurological symptoms of Niemann-Pick disease type C (NPC) in adults and children 2 years and older.
🧬 Where this information comes from
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
Arimoclomol (Miplyffa) is a specialist medicine, on the U.S. market since about 2024, taken with miglustat for the neurological symptoms of Niemann-Pick disease type C, a rare inherited condition, in people 2 years and older; how it helps is not known. Experience with it is still limited, and the side effects people notice most are cold-like symptoms, diarrhea, poorer appetite and weight loss. Watch for an allergic reaction, hives or swelling of the face, lips or throat (angioedema), which showed up within the first two months, and stop it and call the doctor if it happens. It may also harm an unborn baby, so raise pregnancy plans before starting.
Clinical pearls
- It nudges creatinine up without changing kidney filtering, so kidney checks should rely on cystatin C, BUN or measured GFR.
- It blocks the kidney transporter OCT2, so every new prescription should be screened for drugs it could push to higher levels.
- If swallowing is hard, the capsule can be opened and mixed into water, apple juice or soft food.
- Seizures were reported in the clinical trial, so report any new or worsening seizures to the care team promptly.
Patient Information Guide A plain-language walkthrough of Arimoclomol, written from its FDA label.
What Arimoclomol Is Used For
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Arimoclomol is used for one condition.
- Miplyffa (capsule, by mouth) is used together with miglustat to treat neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children 2 years and older.
How Arimoclomol Works
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The way arimoclomol works in NPC is not known. It does reach the spinal fluid in a dose-related way, and it blocks the OCT2 transporter in the kidneys.
Arimoclomol Dosage
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Miplyffa is taken by mouth, usually three times a day, together with miglustat. Dose depends on body weight. People with moderate to severe kidney impairment take it twice a day.
- Take with or without food.
- Swallow capsules whole, or open and mix into water, apple juice or soft food if needed.
- If you miss a dose, skip it and take the next one on schedule.
Follow your prescriber and the label.
Dosing details from the FDA-approved label
From the Arimoclomol prescribing information (“Dosage and Administration”). Doses are set by your prescriber for your product, condition, kidney function and age — never change a dose on your own.
- Patients with actual body weight of 8 kg to 15 kg (in combination with miglustat)
- 47 mg three times a day
- Administer with or without food; for eGFR ≥ 15 to < 50 mL/minute, 47 mg two times a day; same as normal renal function dosage if eGFR ≥ 50 mL/minute
- Patients with actual body weight of > 15 kg to 30 kg (in combination with miglustat)
- 62 mg three times a day
- Administer with or without food; for eGFR ≥ 15 to < 50 mL/minute, 62 mg two times a day; same as normal renal function dosage if eGFR ≥ 50 mL/minute
- Patients with actual body weight of > 30 kg to 55 kg (in combination with miglustat)
- 93 mg three times a day
- Administer with or without food; for eGFR ≥ 15 to < 50 mL/minute, 93 mg two times a day; same as normal renal function dosage if eGFR ≥ 50 mL/minute
- Patients with actual body weight of > 55 kg (in combination with miglustat)
- 124 mg three times a day
- Administer with or without food; for eGFR ≥ 15 to < 50 mL/minute, 124 mg two times a day; same as normal renal function dosage if eGFR ≥ 50 mL/minute
Read the label’s full dosing text
2 DOSAGE AND ADMINISTRATION 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 MIPLYFFA, in combination with miglustat, for patients with an actual body weight of [see Use in Specific Populations ( 8.6 ) and Clinical Pharmacology ( 12.3 )] : 8 kg to 15 kg, is 47 mg two times a day > 15 kg to 30 kg, is 62 mg two times a day > 30 kg to 55 kg, is 93 mg two times a day > 55 kg, is 124 mg two times a day Administer MIPLYFFA with or without food. 2.3 Preparation and Administration Instructions Swallow MIPLYFFA whole. However, for patients who have difficulty swallowing capsules, administer MIPLYFFA in one of two ways: Oral Administration Carefully open the capsule and sprinkle the entire contents into 15 mL of water or apple juice or 15 mL of soft food (e.g., applesauce, pudding, or yogurt). Stir the mixture for 15 seconds. Consume the entire mixture immediately. Feeding Tube Administration (nasogastric or gastric tube) Carefully open the capsule and sprinkle the entire contents into 20 mL of water.
Excerpted — the section continues on the label. Read the full Dosage and Administration section on DailyMed →
Arimoclomol Side Effects
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The most common side effects in the trial were:
Common side effects
- Upper respiratory tract infection (cold-like symptoms)
- Diarrhea
- Weight loss
- Decreased appetite
- Tremor (shakiness)
- Headache
- Hives (urticaria)
When to get medical help
- Call your doctor right away if you get hives, swelling of the face, lips or throat (angioedema), or other signs of an allergic reaction. These happened within the first two months in some patients.
- Stop the medicine and get help if an allergic reaction occurs. Your doctor will decide how to proceed.
- Tell your doctor if you become pregnant or think you might be.
- Seizures were reported in the clinical trial. Report any new or worse seizures to your care team.
Hives with swelling (angioedema) were serious in some patients. Contact your doctor right away if they occur. Low platelets (thrombocytopenia) were also seen in a few patients, mostly those already taking miglustat.
Arimoclomol Warnings and Precautions
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- Allergic reactions: hives and angioedema have occurred, mostly in the first two months. Stop Miplyffa and get care.
- Pregnancy: may cause fetal harm based on animal studies.
- Creatinine: blood levels rise 10 to 20% without harming kidney filtering. Other kidney tests should be used.
- Fertility: may reduce fertility in animal studies.
Arimoclomol Interactions
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Arimoclomol can raise levels of some other medicines.
- OCT2 substrate drugs: Miplyffa blocks OCT2, so these can build up. Your doctor may lower their dose and watch for side effects.
Interactions listed in the FDA-approved label
From the Arimoclomol prescribing information (“Drug Interactions”): what the manufacturer studied or reported to the FDA.
- Substrates of the organic cationic transporter 2 (OCT2 substrates): Arimoclomol is an inhibitor of the OCT2 transporter and may increase the exposure of drugs that are OCT2 substrates. Monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Read the label’s full interactions text
7 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.
Not a complete list. The label covers the interactions the manufacturer studied or reported, and our own interaction database does not cover every medicine either. Bring your full medication list, including vitamins and herbal products, to your pharmacist.
Before Taking Arimoclomol
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- No contraindications are listed.
- Pregnancy: possible fetal harm; discuss pregnancy planning and prevention.
- Breastfeeding: no data; weigh benefits and risks with your doctor.
- Children: studied in those 2 and older; not established under 2.
- Older adults: not studied in people 65 and older.
- Kidney: lower frequency for eGFR 15 to under 50; not studied under 15.
- Liver: not studied in severe impairment.
What Arimoclomol Does in the Body
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In healthy volunteers, arimoclomol caused a reversible rise in serum creatinine of about 19% without changing kidney filtering or blood flow. At up to three times the maximum recommended dose, it did not meaningfully affect the heart's QTc interval on ECG.
How Your Body Processes Arimoclomol
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Arimoclomol reaches its peak blood level in about 30 minutes, and food does not meaningfully change absorption. Its half-life is about 4 hours. It is broken down by several pathways and cleared mostly in urine, with about 42% of a dose leaving unchanged. Moderate to severe kidney impairment roughly doubles exposure.
How to Store Arimoclomol
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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.
Written from the FDA-approved label with AI and reviewed by our pharmacy team. How we use AI →
Arimoclomol: Pharmacist Answers to Common Questions
Quick, plain-English answers to the questions patients ask most about Arimoclomol — the kind of thing our pharmacy team would talk through with you at the counter.
What is this medicine for?
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How do I take it?
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What if I miss a dose?
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What side effects should I expect?
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Will it affect my kidney blood tests?
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Is it safe during pregnancy?
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Is Arimoclomol available over the counter?
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When was Arimoclomol first available?
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Answers drafted from the FDA-approved label with AI and reviewed by our pharmacy team. How we use AI →
More About Arimoclomol on HelloPharmacist
Arimoclomol Forms and Strengths (How It Comes)
Arimoclomol is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Need a version that does not list lactose, a gluten source, a dye or gelatin? Inactive ingredients differ by manufacturer: see Arimoclomol inactive ingredients by manufacturer.
Capsule
How this form is used
- Miplyffa is used with miglustat to treat neurological manifestations of Niemann-Pick disease type C in people 2 years and older.
- Miplyffa can be taken with or without food.
- Swallow Miplyffa capsules whole when possible.
- If swallowing is hard, Miplyffa capsule contents can be mixed into water, apple juice or soft food and taken right away.
- Miplyffa can also be given through a feeding tube mixed with water only; do not save the mixture.
Arimoclomol on the U.S. Market: Products, Makers and Brands
How Arimoclomol appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
1 company lists 4 Arimoclomol product records with the FDA, mostly as capsule; the earliest marketing or approval year on record is 2024. Brand names on the directory include Miplyffa; the rest are generics.
Also listed with the FDA, not a patient dose form: Unfinished capsule (4) — bulk drug substance and unfinished product sold to compounders and other manufacturers to finish, and multi-item kits. These aren’t counted in the dose-form total above.
Compare Arimoclomol Products
A representative set of FDA-listed product records for Arimoclomol — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Capsule | ||||
| 47 mg | Oral | Acer Therapeutics Inc. | NDA | View NDC → |
| 62 mg | Oral | Acer Therapeutics Inc. | NDA | View NDC → |
| 93 mg | Oral | Acer Therapeutics Inc. | NDA | View NDC → |
| 124 mg | Oral | Acer Therapeutics Inc. | NDA | View NDC → |
Showing 4 of 4 products — FDA National Drug Code Directory. See every product, package size and price: browse all 4 Arimoclomol NDCs →
Arimoclomol Side Effects Reported to the FDA (FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Arimoclomol — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Arimoclomol in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
Arimoclomol FDA Approval History
How Arimoclomol went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
Arimoclomol Recall History
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
The FDA’s enforcement reports list no Arimoclomol recalls since July 2021.
✅No Arimoclomol recalls on record since July 2021Source: FDA enforcement (recall) reports, via openFDA; our copy was last updated Oct 8, 2026.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2694283 1 dose form · 4 strengths
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Dose form (SCDF) Oral Capsule RxCUI 2694287In current FDA listings
Look up RxCUI 2694283 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
Arimoclomol Supply and Shortage Status
Whether Arimoclomol is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
Arimoclomol Brand Names
Arimoclomol is sold under one brand name in the FDA directory — Miplyffa. A brand and its generic contain the same active ingredient; the brand name belongs to one company's product.
Who Makes Arimoclomol: Manufacturers and Labelers
One company lists Arimoclomol products with the FDA. By number of product records, the largest are Acer Therapeutics Inc.. Labelers include manufacturers and repackagers; the product in your bottle depends on which one your pharmacy stocks.
Arimoclomol Drug Class (RxNorm)
Arimoclomol belongs to the Other nervous system drugs class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
Sources for This Arimoclomol Page
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How this page is built
HelloPharmacist combines public FDA and NLM records and does not author the underlying clinical facts. The plain-language sections are written with AI from the FDA-approved label text and reviewed by our pharmacy team before publication. Acer Therapeutics Inc. is the representative DailyMed label linked for verification and dates. This is general education, not medical advice: always talk to your doctor or pharmacist about your own medicines. How we build and review drug pages →