Opfolda 65 mg hard capsules

Package leaflet: Information for the user

Introduction

Package leaflet: information for the patient

Opfolda 65 mg hard capsules

miglustat

Read the entire 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 have any questions, ask your doctor, pharmacist or nurse.
• This medicine has been prescribed for you only, and you should not give it to other people, even if they have the same symptoms as you, because it may harm them.
• If you experience any adverse reactions, consult your doctor, pharmacist or nurse, even if they are adverse reactions not listed in this leaflet. See section 4.

Contents of the leaflet

1. What Opfolda is and what it is used for
2. What you need to know before taking Opfolda
3. How to take Opfolda
4. Possible side effects
5. How to store Opfolda
6. Contents of the pack and other information

1. What Opfolda is and what it is used for

What Opfolda is

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

What it is used for

Opfolda is always used in combination with another medicine called “cipaglucosidase alfa”, a type of enzyme replacement therapy (ERT). Therefore, it is very important that you also read the cipaglucosidase alfa package leaflet.

If you have any questions about these medicines, 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 regulate glycogen levels (a type of carbohydrate) in the body.

In Pompe disease, large amounts of glycogen accumulate in the muscles throughout the body. This impairs proper muscle function, such as the muscles involved in walking, the muscles involved in breathing in the lungs, and the heart muscle.

Opfolda binds to cipaglucosidase alfa during treatment. This makes the form of cipaglucosidase alfa more stable, thereby facilitating its uptake from the blood into the muscle cells affected by Pompe disease. Once inside the cells, cipaglucosidase alfa acts like GAA, promoting the breakdown of glycogen and regulating its levels.

2. What you need to know before starting to take Opfolda

Do not take Opfolda

• If you are allergic to miglustat or to any of the other ingredients of this medicine (listed in section 6).
• If you are allergic to alfa cypaglucosidase.

Warnings and precautions

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

Pay attention to serious adverse reactions

Opfolda is used together with cipaglucosidase alfa, a type of enzyme replacement therapy (ERT), so you should also read the package leaflet for cipaglucosidase alfa. These medicines may cause adverse reactions that you must report to your doctor immediately, for example allergic reactions. Signs of an allergic reaction are listed in section 4 “Allergic reactions”.

These reactions may be serious and may occur during or in the hours following administration of the medicine.

Tell your doctor or nurse immediately if you experience or think you may be experiencing an infusion-related reaction or an allergic reaction. Before taking Opfolda, inform your doctor or nurse if you have ever had such a reaction with another ERT.

Children and adolescents

This medicine should not be given to patients under 18 years of age, as the effects of Opfolda in combination with cipaglucosidase alfa are unknown in this age group.

Other medicines and Opfolda

Tell your doctor or nurse if you are taking, have recently taken, or might need to take any other medicines, including medicines obtained without a prescription and herbal medicines.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.

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

• Do not take Opfolda or receive cipaglucosidase alfa if you are pregnant. Inform your doctor immediately if you become pregnant, think you may be pregnant, or plan to become pregnant. There may be risks to the unborn baby.
• Opfolda in combination with cipaglucosidase alfa should not be administered to women who are breastfeeding. A decision must be made whether to discontinue breastfeeding or to discontinue treatment.

Contraception and fertility

Female patients of childbearing potential must use effective contraceptive methods during treatment and for 4 weeks after stopping administration of both medicines.

Driving and using machines

The influence of Opfolda on the ability to drive and use machines is negligible or none. You should also read the package leaflet for cipaglucosidase alfa, as this medicine may affect the ability to drive and use machines.

3. How to take Opfolda

Follow exactly the instructions for administration of this medicine given by your doctor. If you are unsure, consult your doctor or pharmacist again.

Amount of Opfolda to be taken

• Opfolda capsules (miglustat) must be used in combination with cipaglucosidase alfa. Please also refer to the package leaflet for cipaglucosidase alfa.
• If you weigh 50 kg or more, the recommended dose is 4 capsules, each containing 65 mg of miglustat.
• If you weigh between 40 kg and 50 kg, the recommended dose is 3 capsules.

How often to take Opfolda

• You will receive Opfolda and cipaglucosidase alfa once every two weeks. Both are administered on the same day.
• Follow exactly your doctor's instructions for administering both medicines, see Figure 1. This way, the treatment can work as effectively as possible.

Taking Opfolda with food

You must take Opfolda orally on an empty stomach.

• You must fast for 2 hours before and 2 hours after taking this medicine.
• During this 4-hour fasting period, you may drink water, fat-free (skimmed) cow's milk, and tea or coffee. Do not drink cream, whole or semi-skimmed cow's milk, plant-based milks, sugar, or sweeteners. You may add skimmed cow's milk to tea or coffee.
• Two hours after taking Opfolda, you may resume eating and drinking normally.

Figure 1. Chronological schedule of dosing

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

Switching from another enzyme replacement therapy (ERT)

If you are currently receiving another ERT:

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

If you take more Opfolda than you should

Inform your doctor immediately or go to hospital if you accidentally take more capsules than prescribed. You may have an increased risk of experiencing side effects with this medicine (see section 4). Your doctor will provide any symptomatic treatment you may need.

If you forget to take Opfolda

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

If you stop taking Opfolda

Talk to your doctor if you wish to stop treatment with Opfolda. Your disease symptoms may worsen if you discontinue treatment.

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everyone will experience them.

Opfolda is used in combination with cipaglucosidase alfa, and either of these medicines may cause adverse effects.

The following adverse effects may occur:

Allergic reactions

Allergic reactions may cause symptoms such as rash anywhere on the body, swelling of the eyes, prolonged breathing difficulty, cough, swelling of the lips, tongue or throat, skin itching and hives.

Inform your doctor or nurse immediately if you experience or think you may be 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)

• Difficulty breathing (dyspnea)
• Sudden redness of the face, neck or upper chest
• Increased blood pressure
• Stomach pain
• Abdominal swelling
• Flatulence or gas
• Diarrhea, loose stools
• Constipation
• Vomiting
• Fatigue
• Nausea
• Fever
• Itchy rash (urticaria)
• Itchy rash, urge to scratch (pruritus)
• Chills
• Muscle cramps, pain or weakness
• Tremors in one or more parts of the body
• Increased sweating
• Pain
• Altered sense of taste

Uncommon (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
• Malaise, general feeling of lethargy
• Nervousness
• Swelling of the hands, feet, ankles, legs
• Feeling of constant tiredness
• Unusual paleness of the skin
• Low blood pressure
• Decreased platelets or a type of white blood cell (detected in laboratory tests)
• Joint pain
• Pain in the area between the hip and ribs
• Muscle fatigue
• Increased muscle stiffness
• Somnolence
• Pain on one or both sides of the head, stabbing pain, aura, eye pain, sensitivity to light (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 leaflet. You may also report them directly through the national reporting system included in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Opfolda

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

Do not use this medicine after the expiry date stated on the vial and outer carton after "EXP". The expiry date refers to the last day of the month indicated.

No special storage conditions are required.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines and containers you no longer need. This will help protect the environment.

6. Contents of the pack and other information

Composition of Opfolda

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

Capsule contents

Pregelatinized starch (corn)

Magnesium stearate (E470b)

Microcrystalline cellulose (E460i)

Sucralose (E955)

Colloidal silicon dioxide

Capsule shell

Gelatin

Titanium dioxide (E171)

Black iron oxide (E172)

Printing ink (edible)

Black iron oxide (E172)

Potassium hydroxide (E525)

Propylene glycol (E1520)

Strong ammonia solution (E527)

Shellac (E904)

Appearance of the product and contents of the pack

Bottles containing 4 and 24 capsules.

Only some pack sizes may be marketed.

Hard capsule of size 2 with grey opaque cap and white opaque body, printed in black with "AT2221" 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
Tel: +353 (0) 1 588 0836
Fax: +353 (0) 1 588 6851
Email: [email protected]

Manufacturer responsible for batch release

Manufacturing Packaging Farmaca (MPF) B.V.
Neptunus 12, Heerenveen, 8448CN, The Netherlands

For further information about this medicine, please contact the local representative of the Marketing Authorization Holder:

Date of the most recent review of this leaflet:

Other sources of information

Detailed information on this medicinal product 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 medicinal products.