Miglustat: uses, dosing, side effects & brands
Miglustat is a medicine sold in the U.S. under 2 brand and generic names, for gaucher disease. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
Key facts
- Treats
- Gaucher Disease
- Available as
- Capsule
- Sold as
- 2 products — Opfolda and Zavesca
- Prescription?
- Prescription only
- Generic available?
- Not in our catalog
How miglustat is dosed
From the FDA label for Zavesca (application NDA021348). Other miglustat products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
Recommended dosage is 100 mg administered orally three times a day at regular intervals ( 2.1 ). May reduce dosage to 100 mg once or twice a day in some patients due to tremor or diarrhea ( 2.1 ). Adjust in patients with renal impairment ( 2.2 ): Renal Impairment Adjusted Creatinine Clearance (in mL/min/1.73 m 2 ) Recommendations Mild 50 – 70 Start dose at 100 mg twice a day Moderate 30 – 50 Start dose at 100 mg once a day Severe <30 Use is not recommended 2.1 Instructions for Administration Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease. The recommended dose for the treatment of adult patients with type 1 Gaucher disease is one 100 mg capsule administered orally three times a day at regular intervals. If a dose is missed, the next miglustat capsule should be taken at the next scheduled time. It may be necessary to reduce the dose to one 100 mg capsule once or twice a day in some patients due to adverse reactions, such as tremor or diarrhea. 2.2 Patients with Renal Insufficiency In patients with mild renal impairment (adjusted creatinine clearance 50–70 mL/min/1.73 m 2 ), initiate miglustat treatment at a dose of 100 mg twice per day. In patients with moderate renal impairment (adjusted creatinine clearance of 30–50 mL/min/1.73 m 2 ), initiate miglustat treatment at a dose of one 100 mg capsule per day. Miglustat is not…
Miglustat side effects
The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy [see Warnings and Precautions (5.1) ] Tremor [see Warnings and Precautions (5.2) ] Diarrhea and Weight Loss [see Warnings and Precautions (5.3) ] Reductions in Platelet Count [see Warnings and Precautions (5.4) ] The most common adverse reactions (incidence ≥5%) are: diarrhea, weight loss, stomach pain, gas, nausea and vomiting headache including migraine, tremor, leg cramps, dizziness, weakness, vision problems, thrombocytopenia, muscle cramps, back pain, constipation, dry mouth, heaviness in arms and legs, memory loss, unsteady walking, anorexia, indigestion, paresthesia, stomach bloating, stomach pain not related to food, and menstrual changes ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact CoTherix at 1-855-246-5637 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 data described below reflect exposure of 80 patients with type 1 Gaucher disease in two open-label, uncontrolled, monotherapy trials, one open-label, active-controlled trial, and two extensions, who…
Who shouldn’t take miglustat
None. None ( 4 )
Miglustat drug interactions
While co-administration of miglustat 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 miglustat and imiglucerase may lead to increased clearance of imiglucerase ( 7 ).
Every miglustat product we track (2)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
| # | Drug | Rating | Type | Form | Generic? | Pharmacy pays | |
|---|---|---|---|---|---|---|---|
| 1 | Not yet rated | Prescription | Capsule | No data | — | View → | |
| 2 | Not yet rated | Prescription | Capsule | No data | — | View → |
What miglustat pills look like
Imprint codes, colour and shape from the FDA’s labelling data. Match the imprint on your pill — or search any imprint.
How long miglustat keeps
No miglustat label we read sets a separate limit for after opening, but they do specify how it must be stored — and the stability behind any date assumes those conditions.
Does miglustat expire? The in-use limits and storage rules from its labelsWhat people report to the FDA about miglustat
The FDA Adverse Event Reporting System (FAERS) collects reports from patients and clinicians. It holds 210 reports naming miglustat, and the FDA flagged 81% 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:
- diarrhoea31 reports
- pneumonia24 reports
- seizure24 reports
- pyrexia14 reports
- vomiting12 reports
- pneumonia aspiration11 reports
- niemann-pick disease10 reports
- weight decreased10 reports
Read these as a signal, not a rate. A report does not mean miglustat 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 miglustat?
Miglustat (miglustat capsules, 100 mg) is a glucosylceramide synthase inhibitor, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids. miglustat is an N-alkylated imino sugar, a synthetic analog of D-glucose.
Can you take miglustat 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 miglustat against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What brand names is miglustat sold under?
We track 2 miglustat-containing products in the U.S.: Opfolda and Zavesca. They are the same active ingredient; they differ in form, manufacturer, price and FDA recall record.
What forms does miglustat come in?
Across the brands we track, miglustat 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 miglustat?
We do not currently list a generic-labelled miglustat 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). Miglustat: uses, dosing, side effects & brands. https://pharmaranks.com/drugs/miglustat
- MLA
- “Miglustat: uses, dosing, side effects & brands.” pharmaranks, 24 July 2026, https://pharmaranks.com/drugs/miglustat.
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 miglustat on DailyMed (NIH) ↗.