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.
Miglustat Dipharma 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 Miglustat Dipharma contains the active substance miglustat which belongs to a group of medicines that affect metabolism. It is used to treat two conditions:
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 Miglustat Dipharma together with food, or to temporarily reduce your dose. In some cases the doctor may prescribe antidiarrhoeal medicines such as loperamide. 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. Male patients should use reliable birth control methods during their treatment with Miglustat treatment.
- Miglustat Dipharma is used to treat mild to moderate type 1 Gaucher disease in adults.
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.
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.
Other medicines and Miglustat Dipharma Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. Miglustat Dipharma is only used when a patient is considered unsuitable for treatment with enzyme replacement therapy.
- Miglustat Dipharma 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. Miglustat Dipharma works by inhibiting the enzyme called 'glucosylceramide synthase' synthesis of most glycosphingolipids.
What you need to know before you take it
e Miglustat Dipharma
Tell your doctor if you are taking medicines containing imiglucerase, which are sometimes used at the same time as Miglustat Dipharma. They may lower the amount of Miglustat Dipharma in your body. Pregnancy, breast-feeding and fertility You should not take Miglustat Dipharma if you are pregnant or thinking of becoming pregnant. Your doctor can give you more information. You must use effective birth control while taking Miglustat Dipharma. Do not breast-feed while you are taking Miglustat Dipharma. Male patients should use reliable birth control methods during their treatment with Miglustat 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 Miglustat Dipharma may make you feel dizzy. Do not drive or use any tools or machines if you feel dizzy.
How to take it
Miglustat Dipharma Do not take Miglustat Dipharma:
- 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 Miglustat Dipharma
- if you suffer from kidney disease
- if you suffer from liver disease Since some patients have had tingling or numbness in the hands and feet, or a decrease in body weight, while taking Miglustat Dipharma, your doctor will perform the following tests before treatment and during treatment with Miglustat Dipharma:
- 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 tests will help the doctor decide whether these effects are due to your disease or other existing conditions, or due to side effects of Miglustat Dipharma (see section 4 for further details).
Always take this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.
- For typer 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 Miglustat Dipharma (see section 4). Your doctor will tell you how long your treatment will last. Miglustat Dipharma can be taken with or without food. You should swallow the whole capsule with a glass of water.
you should If you take more capsules than you were told to, consult your doctor immediately. Miglustat Dipharma has been used in clinical trials at doses ten times higher than the recommended dose: this caused decreases in white blood cells and other side effects similar to those described in section 4.
Possible side effects
directly via Yellow Card Scheme Website: www.mhra.gov.uk/yellowcard 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.
Keep this medicine out of the sight and reach of children. Do not use this medicine after the expiry date which is stated on the blister and the carton after EXP. The expiry date refers to the last day of that month.
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.
How to store it
Miglustat Dipharma
Do not take a double dose to make up for a forgotten dose. Take the next capsule at the usual time.
This medicinal product does not require any special storage conditions.
Do not stop taking Miglustat Dipharma 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 Miglustat Dipharma or they could be due to existing conditions. Your doctor will perform some tests before and during treatment with Miglustat Dipharma to assess this (see section 2). above, please seek medical advice from your doctor as soon as possible. , 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 Miglustat Dipharma treatment to stop the tremor. Very common side effects – may affect more than 1 in 10 people The most common side effects are diarrhoea, " #$ " # $ weight loss and decreased appetite. when you start treatment with Miglustat Dipharma don't worry. People usually stop losing weight as treatment goes on. Common side effects – 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 $ $ $ forgetfulness and reduced libido. Most patients get one or more of these side effects mentioned above, 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 Miglustat Dipharma 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
Contents of the pack and other information
What Miglustat Dipharma contains
- The active substance is miglustat. Each hard capsule contains 100 mg miglustat.
- The other ingredients are magnesium stearate, gelatine, titanium dioxide (E171), printing ink (consisting of black iron oxide (E172), propylene glycol (E1520) potassium hydroxide, shellac). What Miglustat Dipharma looks like and contents of the pack Miglustat Dipharma is a white opaque 100 mg capsule with "DPH02" printed in black on the top and "100" printed in black on the body. The capsules are presented in PCTFE/PVC and Aluminium blister, in pack size of 84 capsules in non-perforated blisters and 84×1 capsules in perforated unit dose blisters. Not all pack sizes may be marketed. Marketing Authorisation Holder Waymade PLC Sovereign House Miles Gray Road Basildon, Essex, SS14 3FR United Kingdom Manufacturer Doppel Farmaceutici S.r.l Via Volturno 48 20089 Quinto dè Stampi – Rozzano (MI) Italy !
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Frequently asked questions about Miglustat Dipharma 100 mg hard capsules
How do I take Miglustat Dipharma 100 mg hard capsules?
Miglustat Dipharma 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 Miglustat Dipharma 100 mg hard capsules?
The active substance in Miglustat Dipharma 100 mg hard capsules is miglustat.
Are there equivalent medicines to Miglustat Dipharma 100 mg hard capsules?
Medicines with the same active substance, strength and form include: Yargesa (miglustat) 100mg Hard Capsules, Zavesca 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 Miglustat Dipharma 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 Miglustat Dipharma 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
Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.
Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1).
Miglustat Dipharma 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 miglustat 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 miglustat should be evaluated on a regular basis (see section 4.4).
There is limited experience with the use of miglustat in Niemann-Pick type C disease patients under the age of 4 years.
Special populations
Elderly
There is no experience with the use of miglustat 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
Miglustat has not been evaluated in patients with hepatic impairment.
Method of administration
Oral use.
Miglustat Dipharma 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, and in many cases resolved during treatment after between 1 and 3 months. 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 miglustat 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. Miglustat has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease.
Effects on spermatogenesis
Male patients should maintain reliable contraceptive methods while taking Miglustat Dipharma. Studies in the rat have shown that miglustat adversely affects spermatogenesis and sperm parameters and reduces fertility (see sections 4.6 and 5.3). Until further information is available, before seeking to conceive, male patients should cease Miglustat Dipharma and maintain reliable contraceptive methods for a further 3 months.
Special populations
Due to limited experience, miglustat 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 Miglustat Dipharma 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 miglustat 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 miglustat 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 miglustat 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 miglustat.
Niemann-Pick type C disease
The benefit of treatment with miglustat 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 miglustat.
Mild reductions in platelet counts without association to bleeding were observed in some patients with Niemann-Pick type C disease treated with miglustat. In patients included in the clinical trial, 40%-50% of patients had platelet counts below the lower limit of normal at baseline. Monitoring of platelet counts is recommended in these patients.
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 Miglustat Dipharma; the benefit/risk balance should be re-assessed on an individual basis for continuation of therapy.
4.5. Interaction with other medicinal products and other forms of interaction
Limited data suggest that co-administration of miglustat 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 miglustat 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 reproductive toxicity, including dystocia (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. Miglustat Dipharma 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). Until further information is available, it is advised that before seeking to conceive, male patients should cease Miglustat Dipharma and maintain reliable contraceptive methods for 3 months thereafter.
Contraceptive measures should be used by women of childbearing potential. Male patients should maintain reliable contraceptive methods while taking Miglustat Dipharma (see sections 4.4 and 5.3).
4.7. Effects on ability to drive and use machines
Miglustat Dipharma 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 trials with miglustat were diarrhoea, flatulence, abdominal pain, weight loss and tremor (see section 4.4). The most common serious adverse reaction reported with miglustat treatment in clinical trials was peripheral neuropathy (see section 4.4).
In 11 clinical trials in different indications 247 patients were treated with miglustat at doses 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 < 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.
Miglustat 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 national reporting system at: Yellow Card Scheme
Website: www.mhra.gov.uk/yellowcard 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. Miglustat 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
Ask anything about Miglustat Dipharma 100 mg hard capsules. The assistant answers only from this leaflet — if the leaflet does not cover it, it will say so. It does not give medical advice.
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.