Sandoz Miglustat: Uses, Side Effects and Dosage

What is Sandoz Miglustat used for?

Sandoz Miglustat is used for:

• the treatment of mild to moderate Type 1 Gaucher disease in adults who cannot use imiglucerase, or enzyme replacement therapy.
• slowing the progression of some of the neurologic symptoms in adults and children 4 years of age and older with Niemann-Pick Type C disease (affecting the brain and nervous system).

How does Sandoz Miglustat work?

Sandoz Miglustat prevents an enzyme called glucosylceramide synthase from working. This reduces the production of fatty substances called glycosphingolipid glucosylceramide in cells.

Type I Gaucher disease is a condition in which there is a build-up of glucosylceramide in certain cells of the body's immune system called macrophages. This results in liver and spleen enlargement, changes in the blood, and bone disease.

In Niemann-Pick Type C disease, glycosphingolipids (fats) build-up in cells in the brain. This can result in problems with eye movement, eye sight (vision), balance, swallowing, speech, and memory, and in seizures (fits).

What are the ingredients in Sandoz Miglustat?

Medicinal ingredients: Miglustat

Non-medicinal ingredients: gelatin, magnesium stearate, povidone, sodium starch glycolate, and titanium dioxide

Sandoz Miglustat comes in the following dosage forms:

Capsule: 100 mg

Do not use Sandoz Miglustat if:

• you are allergic to miglustat or to any of the non-medicinal ingredients in Sandoz Miglustat or any component of the container (see What are the ingredients in Sandoz Miglustat?).
• you are pregnant or planning to get pregnant. Sandoz Miglustat may harm your unborn baby.

To help avoid side effects and ensure proper use, talk to your healthcare professional before you take Sandoz Miglustat. Talk about any health conditions or problems you may have, including if you:

• have or have had kidney problems
• have liver problems
• have stomach or intestine problems, including inflammatory bowel disease (IBD)
• have or have had cataracts (clouding of the lens of your eye)

Other warnings you should know about:

Sandoz Miglustat should only be prescribed by a healthcare professional experienced in the management of patients with Gaucher disease or Niemann-Pick Type C disease.

Pregnancy, Breastfeeding and Fertility:

Female Patients:

• You must not take Sandoz Miglustat if you are pregnant or planning to become pregnant. Sandoz Miglustat can harm your unborn baby. Your healthcare professional will make sure you are not pregnant before you start taking Sandoz Miglustat.
• If you are able to get pregnant you must use an effective method of birth control while you are taking Sandoz Miglustat.

• You should not breastfeed while you are taking Sandoz Miglustat. It is not known if Sandoz Miglustat passes into breastmilk.

  Male Patients:

• You should not father a child while taking Sandoz Miglustat and for three months after taking your last dose.
• You must use effective birth control while you are taking Sandoz Miglustat and for 3 months after your last dose. Your female partner should also use an effective method of birth control while you are taking Sandoz Miglustat and for 3 months after your last dose.
• Sandoz Miglustat can affect your sperm and reduce your fertility.

Blood Tests and Monitoring: Sandoz Miglustat can cause abnormal blood test results. Your healthcare professional will decide when to do blood tests and will interpret the results. They will also monitor the health of your spleen and liver. You should have a neurological exam, to check the health of your brain and nerves before you start taking Sandoz Miglustat. This will be repeated by your healthcare professional periodically during treatment.

Driving and Operating Machinery: Sandoz Miglustat may cause dizziness. Give yourself time after taking Sandoz Miglustat to see how you feel before driving a vehicle or using machinery.

Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.

The following may interact with Sandoz Miglustat:

• There are no known relevant interactions at this time.

How to take Sandoz Miglustat:

• Always take Sandoz Miglustat exactly as your heathcare professional has instructed you. You should check with your heathcare professional if you are unsure.
• Your healthcare professional will decide how long you should take Sandoz Miglustat.
• Your healthcare professional might give you a lower dose if you have kidney problems.
• Sandoz Miglustat capsules should be swallowed whole with water.

• Sandoz Miglustat can be taken with or without food. Your risk of diarrhea may be reduced if you take Sandoz Miglustat between meals.

Usual dose:

Type 1 Gaucher disease:

• Adults 18 years of age and older: 100 mg three times daily taken at regular intervals.

Niemann-Pick Type C disease:

• Adults and adolescents 12 to 17 years of age: 200 mg three times daily.

• Children 4 to 12 years old: Your healthcare professional will decide on the correct dose for your child.

Overdose:

Missed dose:

If you forget to take a dose of Sandoz Miglustat, do not take another dose to make up for the missed dose. Take your next dose at the usual time.

What are possible side effects from using Sandoz Miglustat?

These are not all the possible side effects you may have when taking Sandoz Miglustat. If you experience any side effects not listed here, tell your healthcare professional.

Side effects may include:

• weight loss
• loss of appetite
• dry mouth
• gas (flatulence)
• stomach pain
• indigestion
• nausea
• constipation
• headache
• dizziness, vertigo
• fatigue
• generalized weakness
• flu-like symptoms
• vision changes
• muscle cramps or spasms
• memory problems

Serious side effects and what to do about them

If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

Reporting Side Effects

Storage:

• Store at room temperature between 15-30°C
• Store in the original container
• Do not use after the expiry date stated on the container
• Keep out of the sight and reach of children

If you want more information about Sandoz Miglustat:

• Talk to your healthcare professional
• Find the full product monograph that is prepared for healthcare professionals and includes this Patient Medication Information by visiting the Health Canada website: (https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html; the manufacturer’s website www.sandoz.ca, or by calling 1-800-361-3062.

Last Revised: August 23, 2022