Molecule
Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option. MedsAcross supplies Miglustat for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.
Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review
From the regulator-approved product information. Each section names its source.
Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.
Source: US FDA prescribing information
Type 1 Gaucher disease is caused by a functional deficiency of glucocerebrosidase, the enzyme that mediates the degradation of the glycosphingolipid glucosylceramide. Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. Miglustat helps reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy). In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids.
Source: US FDA prescribing information
Recommended dosage is 100 mg administered orally three times a day at regular intervals. May reduce dosage to 100 mg once or twice a day in some patients due to tremor or diarrhea. Adjust in patients with renal impairment: 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
Source: US FDA prescribing information
None. None
Source: US FDA prescribing information
Peripheral neuropathy: Perform baseline and follow-up neurological evaluations at 6- month intervals in all patients. Tremor: Reduce dose to ameliorate tremor or discontinue treatment if tremor does not resolve within days of dose reduction. Diarrhea and Weight Loss: Evaluate for underlying gastrointestinal disease in patients who do not respond to usual interventions (e.g. diet modification). Reductions in Platelet Count: Mild reductions in platelet counts without association with bleeding were observed in some patients. Monitoring of platelet counts is recommended.
Source: US FDA prescribing information
The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy Tremor Diarrhea and Weight Loss Reductions in Platelet Count 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.
Source: US FDA prescribing information
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. Co-administration of miglustat and imiglucerase may lead to increased clearance of imiglucerase.
Source: US FDA prescribing information
Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F).
Source: US FDA prescribing information
For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.
4 presentations of Miglustat, sorted by availability.
| Medicine | Manufacturer | Register | Availability |
|---|---|---|---|
| Miglustat 100 mg capsuleOralGeneric name | — | US FDA | Available on request |
| Opfolda 65 mg capsuleOral | Amicus Therapeutics | US FDA | Available on request |
| Yargesa 100 mg capsuleOral | Edenbridge Pharmaceuticals | US FDA | Available on request |
| Zavesca 100 mg capsuleOral | Actelion Pharmaceuticals | US FDA | Available on request |
3 brand names across 1 markets.
Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.
Conditions linked to Miglustat in public data. This is not a list of approved uses, see the clinical information.
Yes, on request. MedsAcross lists 4 presentations of Miglustat and sources them for licensed hospitals, importers and physicians against valid import documents.
The catalogue lists 3 brand names for Miglustat, including Opfolda, Yargesa, Zavesca.
Yes. The US FDA first approved Miglustat on 31 July 2003, according to Drugs@FDA.
Yes. Miglustat has a central EU marketing authorisation (status: authorised), according to European Medicines Agency data.
No listed presentation of Miglustat is recorded as needing a cold chain. Storage conditions are shown on each presentation page.
Miglustat is classified under ATC code A16AX06 (Miglustat).
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