OPFOLDA 65 mg Hard Capsules

Зміст інструкції

1. Opfolda 65 mg hard capsules
   1. What is Opfolda 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

Opfolda 65 mg hard capsules

miglustat

Read this leaflet carefully before you start to take this medicine as 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 pass it on to others, even if their symptoms are the same as yours, as it could be harmful.
• If you get any side effects, even those not listed in this leaflet, tell 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 you take 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 is Opfolda and what it is used for

What is Opfolda
Opfolda is a medicine used in 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 with another medicine called “cipaglucosidase alfa”, a type of replacement
enzyme therapy (ERT). Consequently, it is very important
For any doubts about the medicines, consult your doctor or pharmacist.

How Opfolda works
People affected by Pompe disease have low levels of the acid alpha-glucosidase
enzyme (GAA). This enzyme helps to control glycogen levels (a type of carbohydrate) in the body.
In Pompe disease, high amounts of glycogen accumulate in the body's muscles. This
prevents muscles, such as those that help with walking, those under the lungs that help with
breathing, and the heart muscle, from functioning correctly.
During treatment, Opfolda binds to cipaglucosidase alfa making its conformation more stable,
thus facilitating its absorption from the blood into the muscle cells affected
by Pompe disease. Once inside the cells, cipaglucosidase alfa acts like GAA,
helping to break down glycogen and control its levels.

2. What you need to know before taking Opfolda

Do not use Opfolda

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

Warnings and precautions
Talk to your doctor, pharmacist or nurse before taking Opfolda.
Pay attention to serious side effects
Opfolda is used together with alpha-cipaglucosidase, a type of enzyme replacement therapy (ERT),
which can cause side effects that you should immediately inform your doctor about. These include
allergic reactions. The signs of allergic reactions are listed in Section 4 “Allergic reactions”.
These can be serious and may occur when the medicine is administered or
during the following hours.
Immediately inform your doctor or nurse if you experience allergic reactions or infusion-related
reactions or if you think you may be experiencing them. Tell your doctor or nurse before you
are given Opfolda if you have already experienced this type of reaction with another ERT.

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

Other medicines and Opfolda
Tell your doctor or nurse if you are taking, have recently taken or will take any other
medicines. These include over-the-counter medicines, including herbal medicines.

Pregnancy and breastfeeding
If you are pregnant, suspect you are pregnant, are planning to become pregnant or are breastfeeding
do not take this medicine but ask your doctor or
pharmacist for advice immediately.
There is no experience regarding the use of Opfolda in combination with alpha-cipaglucosidase
during pregnancy. Your doctor will discuss the risks and benefits associated with taking these
medicines with you.

• Do not take Opfolda and/or receive alpha-cipaglucosidase if you are pregnant. Make sure to inform your doctor immediately if you become pregnant, think you might be pregnant or are planning to become pregnant. There could be risks to the fetus.
• Opfolda in combination with alpha-cipaglucosidase should not be given to breastfeeding women. A decision must be made whether to discontinue the treatment or discontinue breastfeeding. Contraception and fertilityWomen of childbearing potential must use reliable contraceptive methods during use of these medicines as well as for 4 weeks after discontinuation of both medicines. Driving and operating machineryOpfolda does not or negligibly alters the ability to drive vehicles and operate machinery. may affect your ability to do so.

3. How to take Opfolda

Take this medicine following exactly the instructions of your doctor. If you have doubts about how to take the medicine, consult your doctor or pharmacist.

How much Opfolda to take

• Opfolda (miglustat) capsules must be used with cipaglucosidase alfa. See also
• If your weight is 50 kg or more, the recommended dose is 4 capsules, each containing 65 mg of miglustat.
• If your weight is between 40 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 used on the same day.
• Take both medicines exactly as indicated by your doctor, see Figure 1. This is to favour the best possible effect of the treatment. Opfolda with foodYou 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 can consume water, fat-free cow's milk (skimmed), tea or coffee. Do not use cream, whole/partially skimmed cow's milk, non-dairy types of milk, sugar or sweeteners. You can consume fat-free cow's milk (skimmed) with tea or coffee.
• Two hours after taking Opfolda, you can resume eating (food and beverages) normally. Figure 1. Dose timing

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

• Your doctor will tell you when to stop the other ERT before starting Opfolda.
• Inform your doctor when you have completed your last dose. If you take more Opfolda than you shouldImmediately inform your doctor or go to hospital if you accidentally take more capsules than prescribed. You may be at greater risk of experiencing side effects with this medicine (see section 4). The 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 therapy with miglustat in association with cipaglucosidase alfa as soon as possible.

If you stop taking Opfolda
Talk to your doctor if you want to stop taking Opfolda. The symptoms of your illness 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 with cipaglucosidase alfa and side effects may occur due to either of these medicines.
The following side effects may occur:
Allergic reactions
Allergic reactions can include symptoms such as a rash on any part of the body, swollen eyes, prolonged difficulty breathing, cough, swelling of the lips, tongue or throat, itchy skin and hives.
Tell your doctor or nurse immediately if you are experiencing or think you may be experiencing an allergic reaction. Tell your doctor or nurse if you have had an allergic reaction like this before.

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 disturbance
• Sensation of numbness, tingling, pins and needles (paresthesia)
• Rapid heartbeat
• Low blood pressure
• Shortness of breath
• Diarrhoea
• Feeling unwell (nausea)
• Stomach pain
• Flatulence
• Swelling
• Vomiting
• Constipation
• Hives
• Rash
• Itchy skin
• Excessive sweating
• Painful muscle contractions
• 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
• Inability to keep or maintain balance
• Migraine
• Unusual paleness of the skin
• Asthma
• Stomach discomfort
• Painful contractions of the esophagus
• Mouth pain
• Change in skin color
• Pain in the area between the ribs and hip
• Muscle fatigue
• Muscle stiffness
• Weakness
• Pain in the cheeks, gums, lips, chin
• Feeling agitated
• Chest pain

Reporting of side effects
If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly through 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 the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the vial and the packaging after the
wording “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 in wastewater or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Contents of the pack and other information

What Opfolda contains

• The active ingredient is miglustat. Each hard capsule contains 65 mg of miglustat.
• The other ingredients are: Capsule contentsPregelatinized starch (from corn) Magnesium stearate (E470b) Microcrystalline cellulose (E460i) Sucralose (E955)

Colloidal silicon dioxide

Capsule shell
Gelatin
Titanium dioxide (E171)
Black iron oxide (E172)

Food grade printing ink
Black iron oxide (E172)
Potassium hydroxide (E525)
Propylene glycol (E1520)
Concentrated ammonia solution (E527)
Shellac (E904)

Description of Opfolda and contents of the pack
Bottles of 4 and 24 capsules.
Not all pack sizes may be marketed.
Size 2 hard capsule, with an opaque grey cap and an opaque white body imprinted
“AT2221” in black on the body, containing powder ranging in colour from white to off-white.

Marketing Authorisation 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
e-mail: [email protected]

Manufacturer
Manufacturing Packaging Farmaca (MPF) B.V.
Neptunus 12, Heerenveen, 8448CN, Netherlands
For further information on this medicinal product, contact the local representative of the
Marketing Authorisation Holder:

België/Belgique/Belgien
Amicus Therapeutics Europe Limited
Tél/Tel: (+32) 0800 89172
e-mail: [email protected]

Lietuva
Amicus Therapeutics Europe Limited
Tel: (+370) 8800 33167
El. paštas: [email protected]

България
Amicus Therapeutics Europe Limited
Teл.: (+359) 00800 111 3214
имейл: [email protected]

Luxembourg/Luxemburg
Amicus Therapeutics Europe Limited
Tél/Tel: (+352) 800 27003
e-mail: [email protected]

Česká republika
Amicus Therapeutics Europe Limited
Tel.: (+420) 800 142 207
e-mail: [email protected]

Magyarország
Amicus Therapeutics Europe Limited
Tel.: (+36) 06 800 21202
e-mail: [email protected]

Danmark
Amicus Therapeutics Europe Limited
Tlf.: (+45) 80 253 262
e-mail: [email protected]

Malta
Amicus Therapeutics Europe Limited
Tel: (+356) 800 62674
e-mail: [email protected]

Deutschland
Amicus Therapeutics GmbH
Tel: (+49) 0800 000 2038
E-Mail: [email protected]

Nederland
Amicus Therapeutics BV
Tel: (+31) 0800 022 8399
e-mail: [email protected]

Eesti
Amicus Therapeutics Europe Limited
Tel: (+372) 800 0111 911
e-post: [email protected]

Norge
Amicus Therapeutics Europe Limited
Tlf: (+47) 800 13837
e-post: [email protected]

Ελλάδα
Amicus Therapeutics Europe Limited
Τηλ: (+30) 00800 126 169
e-mail: [email protected]

Österreich
Amicus Therapeutics Europe Limited
Tel: (+43) 0800 909 639
E-Mail: [email protected]

España
Amicus Therapeutics S.L.U.
Tel: (+34) 900 941 616
e-mail: [email protected]

Polska
Amicus Therapeutics Europe Limited
Tel.: (+48) 0080 012 15475
e-mail: [email protected]

France
Amicus Therapeutics SAS
Tél: (+33) 0 800 906 788
e-mail: [email protected]

Portugal
Amicus Therapeutics Europe Limited
Tel: (+351) 800 812 531
e-mail: [email protected]

Hrvatska
Amicus Therapeutics Europe Limited
Tel: (+358) 0800 222 452
e-pošta: [email protected]

Ireland
Amicus Therapeutics Europe Limited
Tel: (+353) 1800 936 230
e-mail: [email protected]

România
Amicus Therapeutics Europe Limited
Tel.: (+40) 0808 034 288
e-mail: [email protected]

Slovenija
Amicus Therapeutics Europe Limited
Tel.: (+386) 0800 81794
e-pošta: [email protected]

Ísland
Amicus Therapeutics Europe Limited
Sími: (+354) 800 7634
Netfang: [email protected]

Slovenská republika
Amicus Therapeutics Europe Limited
Tel: (+421) 0800 002 437
e-mail: [email protected]

Italia
Amicus Therapeutics S.r.l.
Tel: (+39) 800 795 572
e-mail: [email protected]

Suomi/Finland
Amicus Therapeutics Europe Limited
Puh/Tel: (+358) 0800 917 780
sähköposti/e-mail: [email protected]

Κύπρος
Amicus Therapeutics Europe Limited
Τηλ: (+357) 800 97595
e-mail: [email protected]

Sverige
Amicus Therapeutics Europe Limited
Tfn: (+46) 020 795 493
e-post: [email protected]

Latvija
Amicus Therapeutics Europe Limited
Tel: (+371) 800 05391
e-pasts: [email protected]

United Kingdom (Northern Ireland)
Amicus Therapeutics, UK Limited
Tel: (+44) 08 0823 46864
e-mail: [email protected]

Other sources of information
More detailed information on this medicinal product is available on the website of the European Medicines
Agency: http://www.ema.europa.eu. Furthermore, links to other websites on rare diseases
and related therapeutic treatments are provided.