Opfolda

Package leaflet: Information for the patient

Opfolda 65 mg hard capsules

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

• Keep this leaflet. 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. Do not give it to other people, even if their symptoms are the same as yours, because it could be harmful.
• If you experience any side effects, including those not listed in this leaflet, talk to your doctor. See section 4.

Contents of this 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 to treat late-onset Pompe disease in adults.
This medicine contains the active substance "miglustat".

What Opfolda 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 to also read the package leaflet for cipaglucosidase alfa.
If you have any questions about the medicines, please consult your doctor or pharmacist.

How Opfolda works
People affected by Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA). This enzyme helps regulate glycogen levels (a type of carbohydrate) in the body. In Pompe disease, excessive amounts of glycogen accumulate in the body's muscles. This prevents muscles—including those involved in walking, the muscles below the lungs involved in breathing, and the heart muscle—from functioning properly.

During treatment, Opfolda binds to cipaglucosidase alfa, stabilizing its structure and thereby facilitating its uptake from the bloodstream into the muscle cells affected by Pompe disease. Once inside the cells, cipaglucosidase alfa acts like GAA, helping to break down glycogen and regulate its levels.

2. What you need to know before taking Opfolda

Do not use 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 cipaglucosidase alfa.

Warnings and precautions
Talk to your doctor, pharmacist, or nurse before taking Opfolda.
Be aware of serious side effects
Opfolda is used together with cipaglucosidase alfa, a type of enzyme replacement therapy (ERT).
Therefore, you must also read the package leaflet for cipaglucosidase alfa. These medicines can cause side effects that you must report to your doctor immediately. These include allergic reactions. Signs of allergic reactions are listed in section 4, “Allergic reactions”.
These reactions can be serious and may occur when the medicine is administered or in the hours following administration.
Tell your doctor or nurse immediately if you experience allergic or infusion-related reactions, or if you think you might develop them. Inform your doctor or nurse before Opfolda is administered if you have previously experienced such a reaction with another ERT.

Children and adolescents
This medicine must not be given to patients under 18 years of age. This is because the effects of Opfolda in combination with cipaglucosidase alfa in this age group are unknown.

Other medicines and Opfolda
Tell your doctor or nurse if you are taking, have recently taken, or might take any other medicines. This includes over-the-counter medicines, including herbal products.

Pregnancy and breastfeeding
If you are pregnant, think you may be pregnant, planning a pregnancy, or breastfeeding, do not take this medicine but seek immediate advice from your doctor or pharmacist.
There is no experience regarding the use of Opfolda in combination with cipaglucosidase alfa during pregnancy. Your doctor will discuss with you the risks and benefits of taking these medicines.

• Do not take Opfolda and/or receive cipaglucosidase alfa if you are pregnant. Make sure to inform your doctor immediately if you become pregnant, think you might be pregnant, or are planning a pregnancy. There may be risks to the fetus.
• Opfolda in combination with cipaglucosidase alfa must not be given to women who are breastfeeding. A decision must be made whether to discontinue treatment or to discontinue breastfeeding.

Contraception and fertility
Female patients of childbearing potential must use reliable contraceptive methods during treatment with these medicines and for 4 weeks after discontinuation of both medicines.

Driving and using machines
Opfolda has no effect or has a negligible effect on the ability to drive and use machines. However, you should also read the package leaflet for cipaglucosidase alfa, as this medicine may affect such ability.

3. How to take Opfolda

Take this medicine exactly as your doctor has instructed. If you have any doubts about how to take the medicine, consult your doctor or pharmacist.

How much Opfolda to take

• Opfolda capsules (miglustat) are to be used in combination with alglucosidase alfa. See also the package leaflet for alglucosidase alfa.
• If your body weight is 50 kg or more, the recommended dose is 4 capsules, each containing 65 mg of miglustat.
• If your body weight is between 40 and 50 kg, the recommended dose is 3 capsules.

How often to take Opfolda

• You will receive Opfolda and alglucosidase alfa once every two weeks. Both medicines are administered on the same day.
• Take both medicines exactly as directed by your doctor; see Figure 1. This is to ensure the best possible treatment effect.

Taking Opfolda with food
You must take Opfolda orally on an empty stomach.

• Remain fasting 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, tea, or coffee. Do not add cream, whole or partially skimmed cow's milk, non-dairy milk alternatives, sugar, or sweeteners. You may add fat-free (skimmed) cow's milk to tea or coffee.
• Two hours after taking Opfolda, you may resume eating and drinking normally.

Figure 1. Dosing schedule

Switching from another enzyme replacement therapy (ERT)
If you are currently being treated with another ERT:

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

If you take more Opfolda than you should
Contact your doctor immediately or go to the hospital if you accidentally take more capsules than prescribed. You may be at increased risk of experiencing side effects with this medicine (see section 4). Your doctor will provide appropriate supportive care.

If you forget to take Opfolda
If you miss a dose of Opfolda, consult your doctor or nurse. Contact your doctor or nurse immediately to schedule a new appointment for miglustat therapy in combination with alglucosidase 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 stop treatment.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
Opfolda is used in combination with cipaglucosidase alfa, and side effects may occur due to either of these medicines.
The following side effects may occur:

Allergic reactions
Allergic reactions may include symptoms such as a rash anywhere on the body, swollen eyes, prolonged breathing difficulties, cough, swelling of the lips, tongue or throat, skin itching and hives.
Inform your doctor or nurse immediately if you are experiencing or think you may be experiencing an allergic reaction. Inform your doctor or nurse if you have previously 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)

• Severe, potentially life-threatening allergic reaction (anaphylactic reaction)
• Tremor
• Taste disturbances
• Tingling, numbness, pins and needles sensation (paraesthesia)
• Rapid heartbeat
• Low blood pressure
• Shortness of breath
• Diarrhoea
• Feeling unwell (nausea)
• Stomach discomfort
• Flatulence
• Swelling
• Vomiting
• Constipation
• Hives
• Rash
• Itchy skin
• Excessive sweating
• Painful muscle cramps
• Muscle pain
• Muscle weakness
• Joint pain
• Fatigue
• Fever
• Chills
• Swelling of the hands, feet, ankles, legs
• Increased blood pressure

Uncommon (may affect up to 1 in 100 people)

• Allergic reaction
• Difficulty maintaining balance
• Migraine
• Unusual paleness of the skin
• Asthma
• Stomach discomfort
• Painful contractions of the oesophagus
• Mouth pain
• Skin colour changes
• Pain in the area between the ribs and hips
• Muscle fatigue
• Muscle stiffness
• Weakness
• Pain in cheeks, gums, lips, chin
• Restlessness
• Chest pain

Reporting of side effects
If you experience any side effects, including those not listed in this leaflet, talk to your doctor, pharmacist or nurse. You can also report side effects directly via the national reporting system listed in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store Opfolda

Keep this medicine out of sight and reach of children.
Do not use this medicine after the expiry date which is stated on the vial and carton after the word “Exp.”. The expiry date refers to the last day of that month.
This medicine does not require any special storage conditions.
Do not dispose of any medicine via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

6. Package contents and other information

What Opfolda contains

• The active substance is miglustat. Each hard capsule contains 65 mg of miglustat.
• The other components are: Capsule contents Pregelatinized starch (from maize) Magnesium stearate (E470b) Microcrystalline cellulose (E460i) Sucralose (E955)

Colloidal silicon dioxide
Capsule shell
Gelatin
Titanium dioxide (E171)
Black iron oxide (E172)
Printing ink for food use
Black iron oxide (E172)
Potassium hydroxide (E525)
Propylene glycol (E1520)
Concentrated ammonia solution (E527)
Shellac (E904)

Description of the appearance of Opfolda and contents of the pack
Bottles containing 4 and 24 capsules.
Not all pack sizes may be marketed.
Size 2 hard capsule with an opaque grey cap and opaque white 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
Manufacturing Packaging Farmaca (MPF) B.V.
Neptunus 12, Heerenveen, 8448CN, Netherlands

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

België/Belgique/Belgien Lietuva
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tél/Tel: (+32) 0800 89172 Tel: (+370) 8800 33167
Email: [email protected] El. paštas: [email protected]

България Luxembourg/Luxemburg
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel.: (+359) 00800 111 3214 Tél/Tel: (+352) 800 27003
Email: [email protected] Email: [email protected]

Česká republika Magyarország
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel.: (+420) 800 142 207 Tel.: (+36) 06 800 21202
Email: [email protected] Email: [email protected]

Danmark Malta
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tlf.: (+45) 80 253 262 Tel: (+356) 800 62674
Email: [email protected] Email: [email protected]

Deutschland Nederland
Amicus Therapeutics GmbH Amicus Therapeutics BV
Tel: (+49) 0800 000 2038 Tel: (+31) 0800 022 8399
E-Mail: [email protected] Email: [email protected]

Eesti Norge
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel: (+372) 800 0111 911 Tlf: (+47) 800 13837
E-post: [email protected] E-post: [email protected]

Ελλάδα Österreich
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel: (+30) 00800 126 169 Tel: (+43) 0800 909 639
Email: [email protected] E-Mail: [email protected]

España Polska
Amicus Therapeutics S.L.U. Amicus Therapeutics Europe Limited
Tel: (+34) 900 941 616 Tel.: (+48) 0080 012 15475
Email: [email protected] Email: [email protected]

France Portugal
Amicus Therapeutics SAS Amicus Therapeutics Europe Limited
Tel: (+33) 0 800 906 788 Tel: (+351) 800 812 531
Email: [email protected] Email: [email protected]

Hrvatska România
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel: (+358) 0800 222 452 Tel.: (+40) 0808 034 288
E-pošta: [email protected] Email: [email protected]

Ireland Slovenija
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel: (+353) 1800 936 230 Tel.: (+386) 0800 81794
Email: [email protected] E-pošta: [email protected]

Ísland Slovenská republika
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Sími: (+354) 800 7634 Tel: (+421) 0800 002 437
Netfang: [email protected] Email: [email protected]

Italia Suomi/Finland
Amicus Therapeutics S.r.l. Amicus Therapeutics Europe Limited
Tel: (+39) 800 795 572 Puh/Tel: (+358) 0800 917 780
Email: [email protected] Sähköposti/Email: [email protected]

Κύπρος Sverige
Amicus Therapeutics Europe Limited Amicus Therapeutics Europe Limited
Tel: (+357) 800 97595 Tfn: (+46) 020 795 493
Email: [email protected] E-post: [email protected]

Latvija United Kingdom (Northern Ireland)
Amicus Therapeutics Europe Limited Amicus Therapeutics, UK Limited
Tel: (+371) 800 05391 Tel: (+44) 08 0823 46864
E-pasts: [email protected] Email: [email protected]

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. In addition, links to other websites on rare diseases and their treatments are provided.