Patient Information Leaflet

Opfolda 65 mg Hard Capsules

miglustat

Read this leaflet carefully before you start taking this medicine, because it contains important information for you.

• Keep this leaflet, as you may need to read it again.
• If you are unsure, consult your doctor, pharmacist or nurse.
• This medicine has been prescribed for you only, and you must not give it to others even if they have the same symptoms as you, as it may harm them.
• If you experience any side effects, consult your doctor, pharmacist or nurse, even if they are not listed in this leaflet. See section 4.

1. What Opfolda is and what it is used for

2. What you need to know before you start taking Opfolda

3. How to take Opfolda

4. Possible side effects

5. Storage of Opfolda

6. Contents of the pack and additional information

What is Opfolda

Opfolda is a medication used for the treatment of late-onset Pompe disease in adults. This medication contains the active ingredient “miglustat”.

What is it used for

Opfolda is used always with another medication called “cipaglucosidasa alfa”, a type of enzyme replacement therapy (ERT). Therefore, it is very important that you also read the prospectus of cipaglucosidasa alfa.

If you have any doubts about these medications, consult your doctor or pharmacist.

How Opfolda works

People with Pompe disease have low levels of an enzyme called acid alpha-glucosidase (GAA). This enzyme helps to regulate the levels of glycogen (a type of carbohydrate) in the body.

In Pompe disease, large amounts of glycogen accumulate in the muscles of the entire body. This prevents the proper functioning of the muscles, for example those that help walking, those that facilitate breathing in the lungs and the cardiac muscle.

Opfolda binds to cipaglucosidasa alfa during treatment. This makes the form of cipaglucosidasa alfa more stable, so that it can be more easily absorbed by the affected muscle cells. Once inside the cells, cipaglucosidasa alfa acts as GAA, promoting the breakdown of glycogen and regulating its levels.

Do not take Opfolda

• If you are allergic to miglustat or any of the other components of this medication (listed in section 6).
• If you are allergic to alpha cypaglucosidase.

Warnings and precautions

Consult your doctor, pharmacist, or nurse before starting to take Opfolda.

Pay attention to severe adverse effects

Opfolda is used in combination with alpha cypaglucosidase, an enzyme replacement therapy (ERT), so you should also read the prospectus for alpha cypaglucosidase. These medications may cause adverse effects that you should immediately report to your doctor, such as allergic reactions. The signs of an allergic reaction are listed in section 4 “Allergic reactions”.

These reactions can be severe and occur during administration of the medication or in the hours that follow.

Inform your doctor or nurse immediatelyif you experience or suspect you are experiencing a reaction associated with infusion or an allergic reaction. Before taking Opfolda, inform your doctor or nurse if you have ever had a reaction of this type with another ERT.

Children and adolescents

This medication should not be administered to patients under 18 years of age, as the effects of Opfolda in combination with alpha cypaglucosidase are unknown in this age group.

Other medications and Opfolda

Inform your doctor or nurse if you are using, have used recently, or may need to use any other medication, including over-the-counter medications and herbal medications.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, or if you think you may be pregnant, consult your doctor or pharmacist before using this medication.

There are no data on the use of Opfolda in combination with alpha cypaglucosidase during pregnancy. Your doctor will explain the risks and benefits of taking these medications.

• Do not take Opfolda or receive alpha cypaglucosidase if you are pregnant. Inform your doctor immediately if you become pregnant, think you may be pregnant, or intend to become pregnant. There may be risks to the fetus.
• Opfolda in combination with alpha cypaglucosidase should not be administered to women who are breastfeeding. It should be decided whether to interrupt breastfeeding or interrupt treatment.

Contraception and fertility

Patients of childbearing age should use effective contraceptive methods during treatment and for 4 weeks after stopping both medications.

Driving and operating machinery

The influence of Opfolda on the ability to drive and operate machinery is negligible or insignificant. You should also read the prospectus for alpha cypaglucosidase, as the medication may affect the ability to drive and operate machinery.

Follow exactly the administration instructions for this medication as indicated by your doctor. In case of doubt, consult your doctor or pharmacist again.

Amount of Opfolda to be taken

• Opfolda (miglustat) capsules should be used with cipaglucosidasa alfa. Also, see the prospectus for cipaglucosidasa alfa.
• If you weigh 50 kg or more, the recommended dose is 4 capsules, each with 65 mg of miglustat.
• If you weigh between 40 kg and 50 kg, the recommended dose is 3 capsules.

How often Opfolda should be taken

• You will receive Opfolda and cipaglucosidasa alfa once every two weeks. Both are administered on the same day.
• Follow exactly the administration instructions for both medications as indicated by your doctor, see figure 1. This way, the treatment can work as best as possible.

Taking Opfolda with food

You should take Opfolda orally with an empty stomach.

• You will have to fast for 2 hours before and 2 hours after taking this medication.
• During this 4-hour fasting period, you can drink water, skimmed cow's milk, and tea or coffee. Do not drink cream, whole milk, or semi-skimmed milk, plant-based milks, sugar, or sweeteners. You can drink skimmed cow's milk with tea or coffee.
• Two hours after taking Opfolda, you can eat and drink normally again.

Figure 1. Chronological development of doses

• Miglustat 65 mg hard capsule should be taken approximately 1 hour and no more than 3 hours before the start of the cipaglucosidasa alfa infusion.

Switch from another enzyme replacement therapy (ERT)

If you are currently receiving another ERT:

• Your doctor will tell you when to stop the other ERT before starting Opfolda.
• Inform your doctor when you received the last dose.

If you take more Opfolda than you should

Inform your doctor immediately or go to the hospitalif you accidentally take more capsules than you have been prescribed. You may increase the risk of experiencing side effects with this medication (see section 4). Your doctor will provide you with symptomatic treatment as needed.

If you forget to take Opfolda

If you forget to take a dose of Opfolda, talk to your doctor or nurse. Contact your doctor or nurse immediately to reschedule the administration of miglustat in combination with cipaglucosidasa alfa as soon as possible.

If you interrupt treatment with Opfolda

Talk to your doctor if you want to interrupt treatment with Opfolda. Your disease symptoms may worsen if you interrupt treatment.

Like all medicines, this medicine may cause side effects, although not everyone will experience them.

Opfolda is used in combination with cipaglucosidase alfa and any of these medicines may cause side effects.

The following side effects may occur:

Allergic reactions

Allergic reactions may cause symptoms such as hives on any part of the body, swelling of the eyes, prolonged difficulty breathing, coughing, swelling of the lips, tongue, or throat, itching on the skin, and urticaria.

Inform your doctor or nurse immediatelyif you experience or suspect you are experiencing an allergic reaction. Inform your doctor or nurse if you have ever had a reaction of this type.

Very common(may affect more than 1 in 10 people)

• Headache

Common(may affect up to 1 in 10 people)

• Dyspnea (difficulty breathing)
• Rapid onset of facial, neck, or upper chest redness
• Increased blood pressure
• Abdominal pain
• Abdominal swelling
• Flatulence or gas
• Diarrhea, loose stools
• Constipation
• Vomiting
• Fatigue
• Nausea
• Fever
• Itchy rash (urticaria)
• Hives with itching, urge to scratch (pruritus)
• Chills
• Muscle cramps, pain, or weakness
• Tremors in one or more parts of the body
• Increased sweating
• Pain
• Alteration of taste

Rare(may affect up to 1 in 100 people)

• Asthma
• Allergic reaction
• Upset stomach
• Indigestion
• Throat pain or irritation
• Painful and abnormal contractions of the throat
• General feeling of fatigue, lethargy
• Nervousness
• Swelling of the hands, feet, ankles, legs
• Constant feeling of fatigue
• Unusual paleness of the skin
• Low blood pressure
• Decreased platelets or a type of white blood cell (detected in blood tests)
• Joint pain
• Pain in the area between the hip and ribs
• Muscle fatigue
• Increased muscle stiffness
• Drowsiness
• Pain on one or both sides of the head, stabbing pain, aura, eye pain, light sensitivity (migraine)
• Skin spots
• Balance disorder

Reporting of adverse effects

If you experience any type of adverse effect, consult your doctor, pharmacist, or nurse, even if it is a possible adverse effect not listed in this prospectus. You can also report them directly through thenational notification system included in theAppendix V. By reporting adverse effects, you can contribute to providing more information on the safety of this medicine.

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date that appears on the bottle and on the box after “CAD”. The expiration date is the last day of the month indicated.

No special storage conditions are required.

Medicines should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the containers and medications that you no longer need. In this way, you will help protect the environment.

Composition of Opfolda

• The active ingredient is miglustat. Each hard capsule contains 65 mg of miglustat.
• The other components are:

Content of the capsules

Pregelatinized maize starch

Magnesium stearate (E470b)

Microcrystalline cellulose (E460i)

Sucralose (E955)

Colloidal silicon dioxide

Capsule coating

Gelatin

Titanium dioxide (E171)

Iron oxide black (E172)

Edible printing ink

Iron oxide black (E172)

Potassium hydroxide (E525)

Propylene glycol (E1520)

Strong ammonia solution (E527)

Shellac (E904)

Appearance of the product and contents of the package

Bottles of 4 and 24 capsules.

Only some package sizes may be marketed.

Hard capsule size 2 with opaque grey cap and opaque white body with "AT2221" printed in black on the body, containing white to off-white powder.

Marketing Authorization Holder

Amicus Therapeutics Europe Limited

Block 1, Blanchardstown Corporate Park

Ballycoolin Road

Blanchardstown, Dublin

D15 AKK1

Ireland

Phone: +353 (0) 1 588 0836

Fax: +353 (0) 1 588 6851

Email: info@amicusrx.co.uk

Responsible for manufacturing

Manufacturing Packaging Farmaca (MPF) B.V.

Neptunus 12, Heerenveen, 8448CN, Netherlands

For more information about this medicine, please contact the local representative of the marketing authorization holder:

Last update of this leaflet:

Other sources of information

More detailed information on this medicine is available on the website of the European Medicines Agency:http://www.ema.europa.eu. There are also links to other websites on rare diseases and orphan medicines.