Pharmacy Guide
Patient leaflets and SmPCs, drug interaction checker, official dosages and NHS pharmacy opening hours — all in one place.
Pharmacy Guide
Patient leaflets and SmPCs, drug interaction checker, official dosages and NHS pharmacy opening hours — all in one place.
Zavesca 100 mg hard capsules
⚠ This medicine appears to have been discontinued
The electronic medicines compendium (emc) no longer publishes a Summary of Product Characteristics for this product, which usually means it is no longer marketed in the UK. The patient leaflet below is kept for reference, but the product may not be available.
If you were prescribed this medicine, other products containing Miglustat may still be available. Do not stop your treatment — ask your pharmacist or GP what to use instead.
What it is and what it is used for
for
Zavesca contains the active substance miglustat which belongs to a group of medicines that affect metabolism. It is used to treat two conditions:
Zavesca is used to treat mild to moderate type 1 Gaucher disease in adults.
In type 1 Gaucher disease, a substance called glucosylceramide is not removed from your body. It starts to build up in certain cells of the body's immune system. This can result in liver and spleen enlargement, changes in the blood, and bone disease. The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Zavesca is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy.
Zavesca is also used to treat progressive neurological symptoms in Niemann-Pick type C disease in adults and in children.
If you have Niemann-Pick type C disease, fats such as glycosphingolipids build up in the cells of your brain. This can result in disturbances in neurological functions such as slow eye movements, balance, swallowing, and memory, and in seizures. Zavesca works by inhibiting the enzyme called 'glucosylceramide synthase' which is responsible for the first step in the synthesis of most glycosphingolipids. 2.
What you need to know before you take it
e Zavesca
Do not take Zavesca if you are allergic to miglustat or any of the other ingredients of this medicine (listed in section 6) Warnings and precautions Talk to your doctor or pharmacist before taking Zavesca if you suffer from kidney disease 1
–
if you suffer from liver disease
Your doctor will perform the following tests before treatment and during treatment with Zavesca: an examination to check the nerves in your arms and legs measurement of vitamin B12 levels monitoring growth if you are a child or adolescent with Niemann-Pick type C disease monitoring of blood platelet counts The reason for these tests is that some patients have had tingling or numbness in the hands and feet, or a decrease in body weight, while taking Zavesca. The tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Zavesca (see section 4 for further details). If you have diarrhoea, your doctor may ask you to change your diet to reduce your lactose and carbohydrate intake such as sucrose (cane sugar), or not to take Zavesca together with food, or to temporarily reduce your dose. In some cases the doctor may prescribe anti-diarrhoeal medicines such as loperamide. Cases of Crohn's disease (an inflammatory disease affecting the gut) have been reported in patients with Niemann-Pick type C disease treated with Zavesca. If your diarrhoea does not respond to these measures, or if you have any other abdominal complaint, consult your doctor. In such case, your doctor may decide to conduct further investigations to determine if there is another cause of your symptoms. Male patients should use reliable birth control methods during their treatment with Zavesca, and for 3 months after finishing treatment. Children and adolescents Do not give this medicine to children and adolescents (below 18 years old) with type 1 Gaucher disease because it is not known if it works in this disease. Other medicines and Zavesca Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines. Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Zavesca. They may lower the amount of Zavesca in your body. Pregnancy, breast-feeding and fertility You should not take Zavesca if you are pregnant or thinking of becoming pregnant. Your doctor can give you more information. You must use effective birth control while taking Zavesca. Do not breast-feed while you are taking Zavesca. Male patients should use reliable birth control methods during their treatment with Zavesca, and for 3 months after finishing treatment. If you are pregnant, breast feeding, think you may be pregnant or planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine. Driving and using machines Zavesca may make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy. Zavesca contains sodium This medicine contains less than 1 mmol sodium (23 mg) per capsule, that is to say essentially 'sodium-free'. 3.
How to take it
Zavesca
2
Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.
For type 1 Gaucher disease: For adults, the usual dose is one capsule (100 mg) three times a day (morning, afternoon and evening). This means a daily maximum of three capsules (300 mg).
For Niemann-Pick type C disease: For adults and adolescents (over 12 years old), the usual dose is two capsules (200 mg) three times a day (morning, afternoon and evening). This means a daily maximum of six capsules (600 mg).
For children less than 12 years old, your doctor will adjust the dose for Niemann-Pick type C disease. If you have a problem with your kidneys you may receive a lower starting dose. Your doctor may reduce your dose, e.g., to one capsule (100 mg) once or twice a day, if you suffer from diarrhoea when taking Zavesca (see section 4). Your doctor will tell you how long your treatment will last. To remove the capsule:
1. 2. 3.
Separate at perforations Peel back paper at arrows Push product through foil
Zavesca can be taken with or without food. You should swallow the whole capsule with a glass of water. If you take more Zavesca than you should If you take more capsules than you were told to, consult your doctor immediately. Zavesca has been used in clinical trials at doses up to 3000 mg: this caused decreases in white blood cells and other side effects similar to those described in section 4. If you forget to take Zavesca Take the next capsule at the usual time. Do not take a double dose to make up for a forgotten dose. If you stop taking Zavesca Don't stop taking Zavesca without talking to your doctor. If you have any further questions on the use of this medicine, ask your doctor or pharmacist. 4.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them. Most serious side effects: Some patients have had tingling or numbness in the hands and feet (seen commonly). They could be signs of peripheral neuropathy, due to side effects of Zavesca or they could be due to existing conditions. Your doctor will perform some tests before and during treatment with Zavesca to assess this (see section 2).
3
If you do get any of these effects, please seek medical advice from your doctor as soon as possible. If you get a slight tremor, usually trembling hands, seek medical advice from your doctor as soon as possible. The tremor often disappears without needing to stop the treatment. Sometimes your doctor will need to reduce the dose or stop Zavesca treatment to stop the tremor. Very common: (may affect more than 1 in 10 people) The most common side effects are diarrhoea, flatulence (wind), abdominal (stomach) pain, weight loss and decreased appetite. If you do lose some weight when you start treatment with Zavesca don't worry. People usually stop losing weight as treatment goes on. Common: (may affect up to 1 in 10 people) Common side effects of treatment include headache, dizziness, paraesthesia (tingling or numbness), abnormal coordination, hypoaesthesia (reduced sensation to touch), dyspepsia (heartburn), nausea (feeling sick), constipation and vomiting, swelling or discomfort in the abdomen (stomach) and thrombocytopenia (reduced levels of blood platelets).The neurological symptoms and thrombocytopenia could be due to the underlying disease. Other possible side effects are muscular spasms or weakness, fatigue chills and malaise, depression, difficulty sleeping, forgetfulness, and less libido. Most patients get one or more of these side effects, usually at the start of treatment or at intervals during treatment. Most cases are mild and disappear quite quickly. If any of these side effects cause problems, consult your doctor. He or she may reduce the dose of Zavesca or recommend other medicines to help control side effects. Reporting of side effects If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the Yellow Card Scheme at: https://yellowcard.mhra.gov.uk/ or search for MHRA Yellow Card in the Google Play or Apple App Store. By reporting side effects you can help provide more information on the safety of this medicine. 5.
How to store it
Zavesca
Keep this medicine out of the sight and reach of children. Do not take this medicine after the expiry date which is stated on the carton after 'EXP'. The expiry date refers to the last day of that month. Do not store above 30C. Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer take. These measures will help protect the environment. 6.
Contents of the pack and other information
What Zavesca contains The active substance is miglustat 100 mg. The other ingredients are: 4
Sodium starch glycollate, Povidone (K30), Magnesium stearate. Gelatin, Titanium dioxide (E171). Black iron oxide (E172), Shellac. What Zavesca looks like and contents of the pack Zavesca is a white 100 mg capsule with "OGT 918" printed in black on the cap and "100" printed in black on the body. Box of 4 blister strips, each blister strip containing 21 capsules providing a total of 84 capsules. Marketing Authorisation Holder: Janssen-Cilag Limited 50-100 Holmers Farm Way High Wycombe Buckinghamshire HP12 4EG UK Manufacturer: Janssen Pharmaceutica NV Turnhoutseweg 30 B-2340 Beerse Belgium
For information in large print, tape, CD or Braille, telephone 0800 7318450. This leaflet was last revised in: November 2023
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Frequently asked questions about Zavesca 100 mg hard capsules
How do I take Zavesca 100 mg hard capsules?
Zavesca 100 mg hard capsules comes as capsule containing 100mg. Always follow the dose your doctor or pharmacist has given you, and read the leaflet that comes with the medicine.
What is the active substance in Zavesca 100 mg hard capsules?
The active substance in Zavesca 100 mg hard capsules is miglustat.
Are there equivalent medicines to Zavesca 100 mg hard capsules?
Medicines with the same active substance, strength and form include: Yargesa (miglustat) 100mg Hard Capsules, Miglustat Dipharma 100 mg hard capsules. They are interchangeable only if your prescriber or pharmacist says so.
Where does this information come from?
This leaflet reproduces the patient information leaflet approved for Zavesca 100 mg hard capsules, as published on the electronic medicines compendium (emc). The version printed inside your medicine’s packaging is the one that applies to you.
Can I get Zavesca 100 mg hard capsules without a prescription?
Whether a medicine is available over the counter or on prescription only depends on its licence. Check the leaflet, or ask your pharmacist — they can tell you straight away.
About this leaflet
The text above reproduces the patient information leaflet approved for this medicine, restructured for easier reading.
- Source: electronic medicines compendium (emc), Datapharm
- Active substance: miglustat
- Official document: view the original leaflet on emc →
⚕For healthcare professionals — Summary of Product Characteristics (SmPC)Full SmPC: dosage, interactions, contraindications, warnings+
4.1. Therapeutic indications
Zavesca is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1).
Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (see sections 4.4, and 5.1).
4.2. Posology and method of administration
Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate.
Posology
Dosage in type 1 Gaucher disease
Adult
The recommended starting dose for the treatment of adult patients with type 1 Gaucher disease is 100 mg three times a day.
Temporary dose reduction to 100 mg once or twice a day may be necessary in some patients because of diarrhoea.
Paediatric population
The efficacy of Zavesca in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. No data are available.
Dosage in Niemann-Pick type C disease
Adult
The recommended dose for the treatment of adult patients with Niemann-Pick type C disease is 200 mg three times a day.
Paediatric population
The recommended dose for the treatment of adolescent patients (12 years of age and above) with Niemann-Pick type C disease is 200 mg three times a day.
Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below:
Body surface area (m2)
Recommended dose
> 1.25
200 mg three times a day
> 0.88 - 1.25
200 mg twice a day
> 0.73 - 0.88
100 mg three times a day
> 0.47 - 0.73
100 mg twice a day
≤ 0.47
100 mg once a day
Temporary dose reduction may be necessary in some patients because of diarrhoea.
The benefit to the patient of treatment with Zavesca should be evaluated on a regular basis (see section 4.4).
There is limited experience with the use of Zavesca in Niemann-Pick type C disease patients under the age of 4 years.
Special populations
Elderly
There is no experience with the use of Zavesca in patients over the age of 70.
Renal impairment
Pharmacokinetic data indicate increased systemic exposure to miglustat in patients with renal impairment. In patients with an adjusted creatinine clearance of 50–70 mL/min/1.73 m2, administration should commence at a dose of 100 mg twice daily in patients with type 1 Gaucher disease and at a dose of 200 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
In patients with an adjusted creatinine clearance of 30–50 mL/min/1.73 m2, administration should commence at a dose of 100 mg once daily in patients with type 1 Gaucher disease and at a dose of 100 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease. Use in patients with severe renal impairment (creatinine clearance < 30 mL/min/1.73 m2) is not recommended (see sections 4.4 and 5.2).
Hepatic impairment
Zavesca has not been evaluated in patients with hepatic impairment.
Method of administration
Zavesca can be taken with or without food.
4.3. Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.
4.4. Special warnings and precautions for use
Tremor
Approximately 37% of patients in clinical trials in type 1 Gaucher disease, and 58% of patients in a clinical trial in Niemann-Pick type C disease reported tremor on treatment. In type 1 Gaucher disease, these tremors were described as an exaggerated physiological tremor of the hands. Tremor usually began within the first month of treatment, and in many cases resolved after 1 to 3 months of continued treatment. Dose reduction may ameliorate the tremor, usually within days, but discontinuation of treatment may sometimes be required.
Gastrointestinal disturbances
Gastrointestinal events, mainly diarrhoea, have been observed in more than 80% of patients, either at the outset of treatment or intermittently during treatment (see section 4.8). The mechanism is most likely inhibition of intestinal disaccharidases such as sucrase-isomaltase in the gastrointestinal tract leading to reduced absorption of dietary disaccharides. In clinical practice, miglustat-induced gastrointestinal events have been observed to respond to individualised diet modification (for example reduction of sucrose, lactose and other carbohydrate intake), to taking Zavesca between meals, and/or to anti-diarrhoeal medicinal products such as loperamide. In some patients, temporary dose reduction may be necessary. Patients with chronic diarrhoea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice. Zavesca has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease.
Cases of Crohn's disease have been reported post-marketing in Niemann-Pick type C disease patients treated with Zavesca. Gastrointestinal disturbances are common adverse events of Zavesca. Therefore, in patients with chronic diarrhoea and/or abdominal pain that do not respond to interventions or in the event of clinical worsening, the possibility of Crohn's disease should be considered.
Effects on spermatogenesis
Reliable contraceptive methods should be maintained while male patients are taking Zavesca and for 3 months following discontinuation. Zavesca should be discontinued and reliable contraception be used for the next 3 months before attempting to conceive (see sections 4.6 and 5.3). Studies in the rat have shown that miglustat adversely affects spermatogenesis and sperm parameters, and reduces fertility (see sections 4.6 and 5.3).
Special populations
Due to limited experience, Zavesca should be used with caution in patients with renal or hepatic impairment. There is a close relationship between renal function and clearance of miglustat, and exposure to miglustat is markedly increased in patients with severe renal impairment (see section 5.2). At present, there is insufficient clinical experience in these patients to provide dosing recommendations. Use of Zavesca in patients with severe renal impairment (creatinine clearance < 30 mL/min/1.73 m2) is not recommended.
Type 1 Gaucher disease
Although no direct comparisons with Enzyme Replacement Therapy (ERT) have been performed in treatment-naive patients with type 1 Gaucher disease, there is no evidence of Zavesca having an efficacy or safety advantage over ERT. ERT is the standard of care for patients who require treatment for type 1 Gaucher disease (see section 5.1). The efficacy and safety of Zavesca has not been specifically evaluated in patients with severe Gaucher disease.
Regular monitoring of vitamin B12 level is recommended because of the high prevalence of vitamin B12 deficiency in patients with type 1 Gaucher disease.
Cases of peripheral neuropathy have been reported in patients treated with Zavesca with or without concurrent conditions such as vitamin B12 deficiency and monoclonal gammopathy. Peripheral neuropathy seems to be more common in patients with type 1 Gaucher disease compared to the general population. All patients should undergo baseline and repeat neurological evaluation.
In patients with type 1 Gaucher disease, monitoring of platelet counts is recommended. Mild reductions in platelet counts without association with bleeding were observed in patients with type 1 Gaucher disease who were switched from ERT to Zavesca.
Niemann-Pick type C disease
The benefit of treatment with Zavesca for neurological manifestations in patients with Niemann-Pick type C disease should be evaluated on a regular basis, e.g. every 6 months; continuation of therapy should be re-appraised after at least 1 year of treatment with Zavesca.
Mild reductions in platelet counts without association to bleeding were observed in some patients with Niemann-Pick type C disease treated with Zavesca. In patients included in the clinical trial, 40%-50% had platelet counts below the lower limit of normal at baseline. Monitoring of platelet counts is recommended in these patients.
Reduced growth in the paediatric population
Reduced growth has been reported in some paediatric patients with Niemann-Pick type C disease in the early phase of treatment with miglustat where the initial reduced weight gain may be accompanied or followed by reduced height gain. Growth should be monitored in paediatric and adolescent patients during treatment with Zavesca; the benefit/risk balance should be re-assessed on an individual basis for continuation of therapy.
Sodium
This medicinal product contains less than 1 mmol sodium (23 mg) per capsule, that is to say essentially 'sodium-free'.
4.5. Interaction with other medicinal products and other forms of interaction
Limited data suggest that co-administration of Zavesca and enzyme replacement with imiglucerase in patients with type 1 Gaucher disease may result in decreased exposure to miglustat (approximate reductions of 22% in Cmax and 14% in AUC were observed in a small parallel-group study). This study also indicated that Zavesca has no or limited effect on the pharmacokinetics of imiglucerase.
4.6. Fertility, pregnancy and lactation
Pregnancy
There are no adequate data from the use of miglustat in pregnant women. Studies in animals have shown maternal and embryo-foetal toxicity, including decreased embryo-foetal survival (see section 5.3). The potential risk for humans is unknown. Miglustat crosses the placenta and should not be used during pregnancy.
Breast-feeding
It is not known if miglustat is secreted in breast milk. Zavesca should not be taken during breast-feeding.
Fertility
Studies in the rat have shown that miglustat adversely affects sperm parameters (motility and morphology) thereby reducing fertility (see sections 4.4 and 5.3).
Contraception in males and females
Contraceptive measures should be used by women of child-bearing potential. Reliable contraceptive methods should be maintained while male patients are taking Zavesca and for 3 months following discontinuation (see sections 4.4 and 5.3).
4.7. Effects on ability to drive and use machines
Zavesca has negligible influence on the ability to drive and use machines. Dizziness has been reported as a common adverse reaction, and patients suffering from dizziness should not drive or use machines.
4.8. Undesirable effects
Summary of the safety profile
The most common adverse reactions reported in clinical studies with Zavesca were diarrhoea, flatulence, abdominal pain, weight loss and tremor (see section 4.4). The most common serious adverse reaction reported with Zavesca treatment in clinical studies was peripheral neuropathy (see section 4.4).
In 11 clinical trials in different indications 247 patients were treated with Zavesca at dosages of 50-200 mg t.i.d. for an average duration of 2.1 years. Of these patients, 132 had type 1 Gaucher disease, and 40 had Niemann-Pick type C disease. Adverse reactions were generally of mild to moderate severity and occurred with similar frequency across indications and dosages tested.
Tabulated list of adverse reactions
Adverse reactions from clinical trials and spontaneous reporting, occurring in >1% of patients, are listed in the table below by system organ class and frequency (very common: ≥ 1/10, common: ≥ 1/100 to < 1/10, uncommon: ≥ 1/1,000 to < 1/100, rare: ≥ 1/10,000 to < 1/1,000, very rare: < 1/10,000). Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Blood and lymphatic system disorders
Common
Thrombocytopenia
Metabolism and nutrition disorders
Very common
Weight loss, decreased appetite
Psychiatric disorders
Common
Depression, insomnia, libido decreased
Nervous system disorders
Very common
Tremor
Common
Peripheral neuropathy, ataxia, amnesia, paraesthesia, hypoaesthesia, headache, dizziness
Gastrointestinal disorders
Very common
Diarrhoea, flatulence, abdominal pain
Common
Nausea, vomiting, abdominal distension/discomfort, constipation, dyspepsia
Musculoskeletal and connective tissue disorders
Common
Muscle spasms, muscle weakness
General disorders and administration site reactions
Common
Fatigue, asthenia, chills and malaise
Investigations
Common
Nerve conduction studies abnormal
Description of selected adverse reactions
Weight loss has been reported in 55% of patients. The greatest prevalence was observed between 6 and 12 months.
Zavesca has been studied in indications where certain events reported as adverse reactions, such as neurological and neuropsychological symptoms/signs, cognitive dysfunction and thrombocytopenia could also be due to the underlying conditions.
Reporting of suspected adverse reactions
Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the Yellow Card Scheme Website: https://yellowcard.mhra.gov.uk/ or search for MHRA Yellow Card in the Google Play or Apple App Store.
4.9. Overdose
Symptoms
No acute symptoms of overdose have been identified. Zavesca has been administered at doses of up to 3000 mg/day for up to six months in HIV positive patients during clinical trials. Adverse events observed included granulocytopenia, dizziness and paraesthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or higher dose.
Management
In case of overdose general medical care is recommended.
💬 Ask about this leaflet
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Answers come from the patient leaflet published on the electronic medicines compendium (emc). They are not medical advice. Ask a pharmacist or your GP if you are unsure. For urgent help call NHS 111, or 999 in an emergency.