Arimoclomol
Arimoclomol is an oral medicine used alongside miglustat to treat the neurological symptoms of Niemann-Pick disease type C (NPC) in adults and children aged 2 and older.
🧬 Where this information comes from Click to expand
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: Patient Information Guide
A plain-language walkthrough of Arimoclomol — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Arimoclomol (brand name Miplyffa) is approved for one condition:
- Niemann-Pick disease type C (NPC): Miplyffa is used in combination with miglustat to treat the neurological manifestations — the brain and nervous system symptoms — of NPC in adults and in children aged 2 years and older. It is not approved as a standalone therapy and must always be paired with miglustat.
⚙️How it works▾
The precise mechanism by which arimoclomol helps in Niemann-Pick disease type C is not yet fully understood. What is known is that after taking the capsule, the drug reaches detectable levels in the cerebrospinal fluid (the fluid surrounding the brain and spinal cord), which is relevant given that NPC is a neurological disease. Research into exactly how it produces its clinical benefit in NPC is ongoing.
💊How it's taken▾
Miplyffa is taken by mouth, three times a day, with or without food. The dose is determined by the patient's actual body weight, and patients with moderate to severe kidney impairment (reduced kidney filtration) may need to take it less frequently — twice a day instead of three times. Swallow the capsule whole; if that is difficult, the contents can be mixed into a small amount of liquid or soft food and taken immediately.
- Do not save mixed doses for later — consume right away
- If a dose is missed, skip it and resume at the next scheduled time
- Always take Miplyffa together with miglustat as prescribed
🤒Side effects — in detail▾
The most common side effects reported in clinical trials (occurring in at least 15% of patients) were:
- Upper respiratory tract infections (colds, runny nose)
- Diarrhea
- Decreased weight
- Decreased appetite
- Tremor (shakiness)
- Hives (urticaria)
- Headache
- Elevated creatinine on blood tests (a lab finding, not necessarily a sign of kidney damage)
Serious reactions requiring prompt medical attention include severe allergic reactions (hives and/or angioedema — swelling under the skin, especially around the face and throat). These occurred within the first two months of treatment in the clinical trial and led to some patients stopping the drug.
⚠️Warnings & precautions▾
- Serious allergic reactions: Hives and angioedema (deep skin swelling) have been reported. Stop Miplyffa and get medical help immediately if these occur. Mild reactions should also be addressed promptly by a healthcare provider.
- Risk to unborn babies (embryofetal toxicity): Animal studies showed harm to developing fetuses. Women who can become pregnant should discuss contraception and family planning with their doctor before starting Miplyffa.
- Elevated creatinine lab values: Miplyffa raises blood creatinine levels by roughly 10–20%, but this is a drug effect on a kidney transport protein — not a sign of actual kidney damage. Doctors should use other tests (such as cystatin C, BUN, or directly measured GFR) to monitor kidney function during treatment. This effect reverses when the drug is stopped.
- Fertility concerns: Animal studies suggest Miplyffa may reduce fertility in both males and females. It is not yet known if this effect is reversible in humans.
🔀What it interacts with▾
Arimoclomol affects how certain other drugs are handled by the kidneys:
- OCT2 substrate drugs: Arimoclomol blocks the OCT2 transporter (a protein in the kidneys that helps clear certain medicines). This can raise blood levels of other drugs that rely on OCT2 to be eliminated — potentially causing side effects from those drugs. Your doctor may need to lower the dose of any OCT2 substrate medicines you take and watch you more carefully.
- Creatinine-based kidney tests: Because Miplyffa raises creatinine independently of actual kidney function, creatinine alone should not be used to assess kidney health while on this medicine — alternative tests should be used instead.
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Pregnancy: Tell your doctor if you are pregnant or could become pregnant. Animal data showed risk of birth defects and pregnancy loss. Contraception is recommended for women of reproductive potential taking Miplyffa.
- Breastfeeding: It is not known whether arimoclomol passes into breast milk or what effects it might have on a nursing infant. Discuss the risks and benefits with your doctor.
- Fertility: Both men and women of reproductive age should be aware that animal studies showed reduced fertility. Whether this applies to humans is not yet known.
- Kidney disease: Patients with moderate to severe kidney impairment (eGFR 15–49 mL/min) need a lower dose. Miplyffa has not been studied in patients with eGFR below 15 mL/min.
- Liver disease: Mild to moderate liver impairment does not significantly change how the body handles Miplyffa. Miplyffa has not been studied in severe liver impairment.
- Children under 2: Safety and effectiveness have not been established in children younger than 2 years old.
- Older adults (65+): Clinical trials did not include patients aged 65 or older, so experience in this group is limited.
📡Pharmacodynamics — effects in the body▾
Miplyffa raises creatinine levels in the blood by roughly 10–20% — not because it damages the kidneys, but because it blocks a kidney transport protein called OCT2 that normally helps clear creatinine. Studies confirmed there was no actual change in how well the kidneys filter blood (glomerular filtration rate). This creatinine increase reverses when the medicine is stopped. Miplyffa does not meaningfully affect the heart's electrical activity (QTc interval) at recommended doses.
🔬Pharmacokinetics — how your body processes it▾
After taking Miplyffa by mouth, peak blood levels are reached in about 30 minutes. Food does not significantly affect absorption, so it can be taken with or without a meal. The drug has a short half-life of about 4 hours and is mostly cleared through the urine — about 77% is excreted in urine and about 12% in stool. Arimoclomol reaches detectable levels in the cerebrospinal fluid (the fluid around the brain and spinal cord). People with moderate to severe kidney impairment clear the drug more slowly — about twice the exposure compared to those with normal kidney function — which is why a lower dose is recommended for that group.
📦How to store it▾
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 Arimoclomol’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
🩺 Pharmacist Counseling Corner
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 exactly is Niemann-Pick disease type C, and how does Miplyffa help? ▾
Can I take Miplyffa without miglustat? ▾
What side effects should I expect, and when should I call a doctor? ▾
I have trouble swallowing capsules — can I open them? ▾
Is it safe to take Miplyffa if I'm pregnant or trying to have a baby? ▾
What if I have kidney problems — can I still take this medicine? ▾
Is Arimoclomol available over the counter? ▾
When was Arimoclomol first available? ▾
Who makes Arimoclomol? ▾
💬 Answers drafted from Arimoclomol’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Arimoclomol 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.
Capsule
How this form is used
- Miplyffa capsules are used in combination with miglustat to treat the neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older
- Miplyffa capsules should be swallowed whole and can be taken with or without food
- Miplyffa capsules may be opened and the contents sprinkled into 15 mL of water, apple juice, applesauce, pudding, or yogurt if swallowing is difficult — consume the entire mixture immediately
- For feeding tube administration, Miplyffa capsule contents should be mixed into 20 mL of water only, administered right away via the tube, then the tube flushed with 5 mL of water
- If a Miplyffa dose is missed, skip it and take the next dose at the regularly scheduled time — do not take two doses at once
📊 Arimoclomol across the U.S. market
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.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
🔍 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 → |
| 47 mg | — | Patheon France S.A.S. | DRUG FOR FURTHER PROCESSING | View NDC → |
| 62 mg | — | Patheon France S.A.S. | DRUG FOR FURTHER PROCESSING | View NDC → |
| 93 mg | — | Patheon France S.A.S. | DRUG FOR FURTHER PROCESSING | View NDC → |
| 124 mg | — | Patheon France S.A.S. | DRUG FOR FURTHER PROCESSING | View NDC → |
Showing 8 of 8 products — FDA National Drug Code Directory. See every product, package size and price: browse all 8 Arimoclomol NDCs →
📈 What people report — real-world data (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.
🏛️ The road to your pharmacy
How Arimoclomol went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 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.
Source: openFDA drug enforcement (recall) reports.
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
-
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.
📦 Supply & 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.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
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.
🔬 Where this comes from
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 we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
For canonical ingredient pages, bounded product-attributed indication and administration excerpts may be added to the AI evidence, and the generator is instructed to preserve available product, brand, route, and form qualifiers instead of treating one label as universal. Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Acer Therapeutics Inc. is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.