Miglustat
Miglustat is an oral capsule medicine used for two rare inherited enzyme disorders: type 1 Gaucher disease and, in combination with Pombiliti, late-onset Pompe disease.
🧬 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 7 clinical label sets 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 →
Miglustat, on the U.S. market since about 2003 and available generically, is a specialist capsule that slows the body's production of a fatty substance people with type 1 Gaucher disease cannot break down well (substrate reduction), for adults who cannot use enzyme replacement, and, as Opfolda, stabilizes the enzyme Pombiliti in late-onset Pompe disease. The gut takes the brunt, with diarrhea, gas and weight loss very common early on, and a shaky-hand tremor is also frequent. Report pain, numbness, tingling or weakness in the hands or feet promptly, since these can signal nerve damage (peripheral neuropathy). Opfolda with Pombiliti must not be used in pregnancy.
Clinical pearls
- For Gaucher dosing, taking the capsules between meals may ease diarrhea, and food slows absorption without reducing the total absorbed.
- Opfolda is taken only every other week, always together with the Pombiliti treatment, never on its own.
- Expect a nerve exam before starting and about every six months, plus blood counts to catch mildly low platelets.
- Kidney function changes how miglustat is cleared, so lower doses may be needed if your kidneys are not working well.
Patient information guide A plain-language walkthrough of Miglustat, written from its FDA label.
What Miglustat is used for
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Miglustat is used for two rare inherited conditions, depending on the product.
- Miglustat, Zavesca and Yargesa (oral capsules): used alone for mild to moderate type 1 Gaucher disease in adults who cannot use enzyme replacement therapy.
- Opfolda (oral capsule): used with Pombiliti for adults with late-onset Pompe disease who weigh at least 40 kg and are not improving on enzyme replacement therapy.
How Miglustat works
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In type 1 Gaucher disease, the body cannot break down a fatty substance called glucosylceramide well enough. Miglustat blocks an enzyme called glucosylceramide synthase, which starts the making of this fat and related fats. With less of it being made, the body's remaining enzyme can work more effectively. This is called substrate reduction therapy.
Miglustat dosage
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Miglustat is taken by mouth as a capsule. Always follow your prescriber and your product label.
- Opfolda is taken every other week, together with Pombiliti. The dose depends on body weight.
- For Gaucher disease, the studies used dosing three times daily.
- Food slows how fast miglustat is absorbed but does not meaningfully change the total amount. Taking it between meals may help with diarrhea.
Dosing details from the FDA-approved label
From the Miglustat 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 weighing ≥50 kg (Opfolda, administered in combination with Pombiliti)
- 260 mg administered orally every other week
- Dosage based on actual body weight
- Patients weighing ≥40 kg to
- 195 mg administered orally every other week
- Dosage based on actual body weight; the label text provided is truncated after '≥40 kg to', so the upper weight bound is not shown in the supplied text
Read the label’s full dosing text
2 DOSAGE AND ADMINISTRATION Verify pregnancy status in females of reproductive potential prior to initiating treatment. (2.1) Administer OPFOLDA in combination with Pombiliti. (2.2) Recommended OPFOLDA dosage (based on actual body weight), administered orally every other week, is: (2.2) 260 mg for patients weighing ≥50 kg. 195 mg for patients weighing ≥40 kg to <50 kg. Start OPFOLDA in combination with Pombiliti 2 weeks after the last ERT dose. (2.2) Take OPFOLDA with an unsweetened beverage approximately 1 hour before the start of Pombiliti infusion; do not consume other beverages or food for at least 2 hours prior to and 2 hours after taking OPFOLDA. (2.2) Missed dose: If the OPFOLDA dosage is missed, Pombiliti should not be administered and treatment should be rescheduled at least 24 hours after OPFOLDA was last taken. If OPFOLDA in combination with Pombiliti are both missed, re-start treatment as soon as possible. (2.2) See full prescribing information for recommended OPFOLDA dosage in patients with renal impairment. (2.3) 2.1 Pregnancy Evaluation Prior to Initiating Treatment Verify the pregnancy status of females of reproductive potential prior to initiating OPFOLDA in combination with Pombiliti [see Use in Specific Populations (8.1, 8.3) ] . 2.2 Recommended Dosage and Administration OPFOLDA must be administered in combination with Pombiliti (see Figure 1 for the dosing timeline). Refer to the Pombiliti Prescribing Information for Pombiliti dosage and administration recommendations. The recommended dosage of OPFOLDA is based on actual body weight. For patients weighing: ≥50 kg, the recommended dosage is 260 mg orally every other week. ≥40 kg to <50 kg, the recommended dosage is 195 mg orally every other week. Start OPFOLDA in combination with Pombiliti 2 weeks after the last ERT dose. Take OPFOLDA approximately 1 hour before intravenous administration of Pombiliti. Swallow the OPFOLDA capsules whole only with unsweetened beverages (e.g., water, tea or coffee with no cream, sugar, or sweeteners). Do not consume other beverages or food for at least 2 hours prior to and 2 hours after administration of OPFOLDA. Missed Dose If the OPFOLDA dosage is missed, Pombiliti should not be administered and treatment should be rescheduled at least 24 hours after OPFOLDA was last taken.
Excerpted — the section continues on the label. Read the full Dosage and Administration section on DailyMed →
Miglustat side effects
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The most common side effects reported in Gaucher disease studies were:
Common side effects
- Diarrhea
- Weight loss
- Gas and stomach pain
- Nausea and vomiting
- Headache
- Tremor (shaky hands)
- Leg or muscle cramps
- Dizziness
- Stomach pain, gas and bloating
- Headache, including migraine
- Vision problems
- Low platelet count
When to get medical help
- Call your doctor if you notice pain, weakness, numbness or tingling in your hands or feet. These can be signs of peripheral neuropathy (nerve damage).
- Call your doctor if you develop a new tremor or an existing tremor gets worse and does not settle down.
- Call your doctor if diarrhea or weight loss is persistent or does not respond to diet changes.
- Tell your doctor if you are pregnant or become pregnant while taking Opfolda with Pombiliti.
Contact your doctor about nerve symptoms (pain, numbness, tingling, weakness), a tremor that persists, or diarrhea and weight loss that do not improve.
Miglustat warnings and precautions
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- Pregnancy (Opfolda with Pombiliti): contraindicated because of possible harm to the unborn baby.
- Peripheral neuropathy: nerve checks are recommended before starting and about every 6 months.
- Tremor: may need a dose reduction or stopping treatment if it does not go away.
- Diarrhea and weight loss: very common. Persistent symptoms should be checked for an underlying bowel disease.
- Platelet counts: mild drops were seen, so monitoring is recommended.
Miglustat interactions
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Few interactions are described.
- Imiglucerase: miglustat may increase its clearance, though the evidence is not conclusive.
- Loperamide: commonly used to treat miglustat-related diarrhea.
- Many common medicines broken down by liver enzymes are unlikely to be affected.
Interactions listed in the FDA-approved label
From the Miglustat prescribing information (“Drug Interactions”): what the manufacturer studied or reported to the FDA.
- Imiglucerase: Co-administration of miglustat capsules appeared to increase the clearance of imiglucerase by 70%, but these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase. Co-administration of YARGESA and imiglucerase may lead to increased clearance of imiglucerase.
Read the label’s full interactions text
7 DRUG INTERACTIONS While co-administration of miglustat capsules appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase [ see Clinical Pharmacology (12.3) ]. Co-administration of YARGESA and imiglucerase may lead to increased clearance of imiglucerase ( 7 ).
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 Miglustat
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- Do not use Opfolda with Pombiliti during pregnancy.
- Females who could become pregnant should have pregnancy status checked before starting Opfolda.
- Breastfeeding is not recommended with Opfolda and Pombiliti.
- Tell your doctor about kidney problems, since lower doses may be needed.
- Tell your doctor about bowel disease such as inflammatory bowel disease.
- Tell your doctor about existing tremor or nerve symptoms.
How your body processes Miglustat
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Miglustat reaches its peak level in the blood about 2 to 2.5 hours after a dose. Its effective half-life is about 6 to 7 hours. It does not bind to proteins in the blood and can cross into the brain's fluid. It is cleared mainly by the kidneys, largely unchanged, which is why kidney problems can raise levels.
How to store Miglustat
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Store at 20°C to 25°C (68°F to 77°F). Keep out of reach of children.
Written from the FDA-approved label with AI and reviewed by our pharmacy team. How we use AI →
Supplements & herbs that interact with Miglustat
17 supplements and herbal products have documented interactions with Miglustat in the licensed clinical database we use. Most are manageable — but your pharmacist should know about everything you take, including vitamins and herbals.
- Major · 1
- Moderate · 7
- Minor · 9
Educational information from a licensed clinical database (Natural Medicines) — see our data sources & update cadence. Not medical advice: an interaction being documented does not mean it will happen to you; do not start or stop medications or supplements without professional guidance.
Miglustat: pharmacist answers to common questions
Quick, plain-English answers to the questions patients ask most about Miglustat — the kind of thing our pharmacy team would talk through with you at the counter.
What is miglustat for?
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Should I take it with food?
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What side effects should I expect?
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When should I call my doctor?
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Can I take it if I'm pregnant or breastfeeding?
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Does it interact with other medicines?
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Is Miglustat available over the counter?
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When was Miglustat first available?
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Who makes Miglustat?
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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 Miglustat on HelloPharmacist
Miglustat forms and strengths (how it comes)
Miglustat 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 Miglustat inactive ingredients by manufacturer.
Capsule
How this form is used
- Miglustat, Zavesca and Yargesa are used alone for mild to moderate type 1 Gaucher disease in adults who cannot use enzyme replacement therapy.
- Opfolda is used with Pombiliti for adults with late-onset Pompe disease who weigh at least 40 kg and are not improving on enzyme replacement therapy.
- Opfolda is taken by mouth every other week, together with Pombiliti. The dose is based on body weight.
- Opfolda requires a pregnancy status check before starting in females who could become pregnant.
Miglustat on the U.S. market: products, makers and brands
How Miglustat 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.
7 companies list 7 Miglustat product records with the FDA, mostly as capsule; the earliest marketing or approval year on record is 2003. Brand names on the directory include Zavesca, Yargesa, Opfolda; the rest are generics.
Also listed with the FDA, not a patient dose form: Powder (7) — bulk drug substance sold to compounders/manufacturers and multi-item kits. These aren’t counted in the dose-form total above.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
How widely Miglustat is used (Medicaid data)
A general measure of how commonly Miglustat is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Miglustat is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Miglustat prescribed? Of the 1,601 medications we measure, Miglustat ranks #1,490 by Medicaid prescriptions — more than 7% of them.
Utilization by Miglustat product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
miglustat 100 MG Oral Capsule
Summed across the 2 of 16 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 401938
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Miglustat, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 2 of 16 listed Miglustat NDCs with Medicaid activity.
Compare Miglustat products
A representative set of FDA-listed product records for Miglustat — 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 | ||||
| 100 mg | Oral | Actelion Pharmaceuticals US, Inc. | NDA | View NDC → |
| 65 mg | Oral | Amicus Therapeutics Us, Llc | NDA | View NDC → |
| 100 mg | Oral | ANI Pharmaceuticals, Inc. | ANDA | View NDC → |
| 100 mg | Oral | CoTherix, Inc. | NDA AUTHORIZED GENERIC | View NDC → |
| 100 mg | Oral | Edenbridge Pharmaceuticals LLC. | ANDA | View NDC → |
| 100 mg | Oral | Navinta LLC | ANDA | View NDC → |
| 100 mg | Oral | Zydus Pharmaceuticals USA Inc. | ANDA | View NDC → |
Showing 7 of 7 products — FDA National Drug Code Directory. See every product, package size and price: browse all 7 Miglustat NDCs →
Miglustat 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 Miglustat — 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 Miglustat 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.
Miglustat FDA approval history
How Miglustat went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
Miglustat 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 Miglustat recalls since July 2021.
✅No Miglustat recalls on record since July 2021Source: FDA enforcement (recall) reports, via openFDA; our copy was last updated Sep 24, 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 402316 1 dose form · 2 strengths
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Dose form (SCDF) Oral Capsule RxCUI 402347In current FDA listings
Look up RxCUI 402316 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.
Miglustat supply and shortage status
Whether Miglustat 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.
Miglustat brand names
Miglustat is sold under 3 brand names in the FDA directory — Zavesca, Yargesa, Opfolda. A brand and its generic contain the same active ingredient; the brand name belongs to one company's product.
Who makes Miglustat: manufacturers and labelers
7 companies list Miglustat products with the FDA. By number of product records, the largest are Amicus Therapeutics Us, Llc, ANI Pharmaceuticals, Inc., Actelion Pharmaceuticals US, Inc.. Labelers include manufacturers and repackagers; the product in your bottle depends on which one your pharmacy stocks.
Miglustat drug class (RxNorm)
Miglustat belongs to the Glucosylceramide Synthase Inhibitor 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 Miglustat 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. CoTherix, 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 →