Zavesca (miglustat)
An approved treatment for Gaucher Disease.
The same compound appears under different names depending on the context. Here is how to identify Miglustat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Miglustat
- Brand name
- Zavesca
- Development code
- OGT 918
- Drug class
- Substrate reduction therapy
- Manufacturer
- Janssen (originally Actelion/Oxford GlycoSciences)
- How it's taken
- Taken orally as a capsule 3 times daily (100 mg each).
The first oral treatment for Type 1 Gaucher disease. Zavesca works by reducing the production of the fatty substance that accumulates in Gaucher patients, offering a pill-based alternative to intravenous enzyme replacement therapy.
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Where Miglustat fits
Oral substrate reduction therapy for adults with mild-to-moderate Gaucher disease type 1 when enzyme replacement is not suitable. Generally considered second-line after eliglustat or ERT.
How Miglustat works
Rather than replacing the missing enzyme (like ERTs do), miglustat takes the opposite approach: it reduces the amount of glucocerebroside being produced in the first place. By inhibiting glucosylceramide synthase, the enzyme that makes glucocerebroside, miglustat reduces the workload on whatever residual enzyme activity the patient has. This is called substrate reduction therapy.
Mechanism: Oral substrate reduction therapy that inhibits glucosylceramide synthase to reduce the production of glucocerebroside
Side effects and safety
Gastrointestinal side effects are very common, including diarrhea (especially in the first few months), flatulence, abdominal pain, nausea, and weight loss. Peripheral neuropathy (numbness and tingling) can occur and requires monitoring. Tremor has been reported. The label also warns about mild drops in platelet count, so platelet counts should be checked during treatment.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Miglustat
Taken orally as a capsule 3 times daily (100 mg each). The oral route is a key advantage over IV enzyme replacement therapies. Approved for adults with mild-to-moderate Type 1 Gaucher disease for whom ERT is not suitable.
Availability and cost
Generic versions may be available at a lower cost. Ask your pharmacist.
Oral substrate reduction therapy, the first oral alternative to IV enzyme replacement for Gaucher disease. Specialty pricing for a rare disease despite being a small molecule.
Clinical trial results
Clinical trials showed significant reductions in liver and spleen volume, with improved platelet counts and hemoglobin levels. Approved as second-line therapy for patients who cannot receive enzyme replacement therapy.
Development history
Approved by FDA in July 2003 for Type 1 Gaucher disease. In Europe and some other countries it is also approved for Niemann-Pick disease type C; in the U.S. it is not approved on its own for that condition, though Miplyffa (arimoclomol) is approved to be taken together with miglustat for NPC. Was the first oral therapy available for Gaucher patients, though ERTs remain first-line for most patients due to greater efficacy.
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Common questions about Miglustat
▸What is Miglustat (Zavesca)?
The first oral treatment for Type 1 Gaucher disease. Zavesca works by reducing the production of the fatty substance that accumulates in Gaucher patients, offering a pill-based alternative to intravenous enzyme replacement therapy.
▸How does Miglustat work?
Rather than replacing the missing enzyme (like ERTs do), miglustat takes the opposite approach: it reduces the amount of glucocerebroside being produced in the first place. By inhibiting glucosylceramide synthase, the enzyme that makes glucocerebroside, miglustat reduces the workload on whatever residual enzyme activity the patient has. This is called substrate reduction therapy.
▸What are the side effects of Miglustat?
Gastrointestinal side effects are very common, including diarrhea (especially in the first few months), flatulence, abdominal pain, nausea, and weight loss. Peripheral neuropathy (numbness and tingling) can occur and requires monitoring. Tremor has been reported. The label also warns about mild drops in platelet count, so platelet counts should be checked during treatment.
▸How is Miglustat taken?
Taken orally as a capsule 3 times daily (100 mg each). The oral route is a key advantage over IV enzyme replacement therapies. Approved for adults with mild-to-moderate Type 1 Gaucher disease for whom ERT is not suitable.
▸Is Miglustat FDA approved?
Yes, Miglustat (Zavesca) is FDA approved (2003) for the treatment of Gaucher Disease.
▸When is miglustat used instead of enzyme replacement therapy?
Miglustat (Zavesca) is approved for adults with mild-to-moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not suitable. It is generally considered second-line after eliglustat or ERT, used when IV infusions are not feasible or preferred.
▸What gastrointestinal side effects should I expect?
Gastrointestinal side effects are very common, especially in the first few months. Diarrhea, flatulence, abdominal pain, nausea, and weight loss are frequently reported. These often improve over time. A lactose-free diet may help manage GI symptoms.
▸Is miglustat also approved for other conditions?
In the U.S., Zavesca is approved only for type 1 Gaucher disease. Miglustat is approved for Niemann-Pick disease type C in Europe and some other countries, and in the U.S. Miplyffa (arimoclomol) is approved to be used together with miglustat for NPC. A different miglustat product, Opfolda (65 mg), is approved to be taken with Pombiliti for late-onset Pompe disease.
▸How does miglustat compare to eliglustat?
Both are oral substrate reduction therapies, but eliglustat (Cerdelga) is generally preferred as it has demonstrated non-inferiority to ERT and has fewer GI side effects. Miglustat is taken 3 times daily versus eliglustat's once or twice daily dosing.
▸What is peripheral neuropathy risk with miglustat?
Peripheral neuropathy (numbness and tingling in hands and feet) can occur with miglustat and requires monitoring. Patients should report any new neurological symptoms to their doctor. Regular neurological assessments are recommended.
▸How is miglustat dosed?
Miglustat is taken orally as a 100 mg capsule 3 times daily. The oral route is a key advantage over IV enzyme replacement therapies, allowing patients to manage their condition at home without infusion visits.