Opfolda® 65 mg hard capsul – patient leaflet, side effects, dosage

What it is used for

Opfolda is always used with ano medicine called ‘cipaglucosidase a type of enzyme replacement th (ERT). It is therefore very importa you also read the package leaflet cipaglucosidase alfa.

illness are the same as yours. If you get any side effects, talk to yo doctor, pharmacist, or nurse. This includes any possible side effects n listed in this leaflet. See section 4.

hat is in this leaflet

. What Opfolda is and what it is used . What you need to know before you t

•

W

1

2

ur

ot

for

ake

If you have any questions about medicines, please ask your doct pharmacist.

How Opfolda works

People with Pompe disease have levels of the enzyme acid alpha-glucosidase (GAA). This enzyme

Opfolda

1. 3. How to take Opfolda
2. 4. Possible side effects
3. 5. How to store Opfolda
4. 6.

   is used

   d in the isease in e active

   1. 2. What you need to know take Opfolda

Do not use Opfolda

• If you are allergic to miglu the other ingredients of th (listed in section 6).

ther

alfa’, erapy nt that of

• If you are allergic to cipag alfa.

Warnings and precaution

Talk to your doctor, pharma before taking Opfolda.

Look out for serious side eff Opfolda is used together wi

your or or

low

helps

cipaglucosidase alfa, a type replacement therapy (ERT), should also read the packa cipaglucosidase alfa. These can cause side effects that tell your doctor about straig includes allergic reactions. reactions are listed in sectio reactions’. These can be se

control levels of glycogen (a type carbohydrate) in the body.

In Pompe disease, high levels of build up in the muscles of the bo keeps muscles, such as the mus help you walk, the muscles unde lungs that help you breathe, and

of

glycogen dy. This cles that r the

the heart

happen when you are being medicine or during the hour

Tell a doctor or nurse imme are experiencing infusion-re reactions or think you may them. Inform your doctor or have ever had any such rea

muscle, from working properly.

Opfolda binds to cipaglucosidas during treatment. This makes the of cipaglucosidase alfa more sta it can be more easily absorbed fr blood by the muscle cells that ar by Pompe disease. When in the cipaglucosidase alfa works like G

e alfa

shape

ble, so

om the

e affected cells,

AA to

another ERT before you are

Children and adolescents

This medicine should not b patients under the age of 1 This is because the effects combination with cipagluco this age group are not know

help break down glycogen and c levels.

ontrol its Other medicines and Opf

Tell a doctor or nurse if you have recently used, or will b other medicines. This inclu obtained without a prescrip herbal medicines.

before you

stat or any of is medicine

Pregnancy and brea

If you are pregnant or you may be pregnant, have a baby, do not ta but talk to your doctor immediately for advic

There is no experienc

lucosidase

s

cist, or nurse

ects

th

use of Opfolda in com cipaglucosidase alfa d Your doctor will discu and benefits of taking

• Do not take Opfolda cipaglucosidase alf Be sure to tell your

of enzyme

so you

ge leaflet of

medicines

you need to ht away. This Signs of allergic n 4 ‘Allergic vere and may

if you get pregnant, be pregnant, or if yo become pregnant. T the unborn baby.

• Opfolda in combina cipaglucosidase alf to women who are b decision will need to to stop treatment or

given the

s after.

diately if you lated or allergic be experiencing nurse if you ction with

feeding.

Contraception and fe

Female patients of chi must use reliable birth during and for 4 week medicines.

Driving and using ma

given Opfolda.

e given to

8 years old.

of Opfolda in sidase alfa in n.

Opfolda has no or neg your ability to drive or should also read the p cipaglucosidase alfa, have an impact.

3. How to take Opfol

olda

are using,

e using any des medicines tion, including

Always take this medi doctor has told you. C doctor or pharmacist i how the medicine sho

st-feeding

breast-feeding, think or are planning to ke this medicine or pharmacist

e.

e with the

How much Opf

• Opfolda (miglu used with cipa the package le alfa.
• If you weigh 5 recommended containing eac

bination with

uring pregnancy. ss with you the risks these medicines.

and / or receive a if you are pregnant. doctor immediately

• If you weigh b the recommen

How often to ta

• You will receiv cipaglucosida week. Both ar
• Take both med

think that you may u are planning to here may be risks to

tion with

a should not be given reast-feeding. A be made whether to stop breast-

have been told Figure 1. This i work as well a

Opfolda with fo

You must take O empty stomach.

• Fast for 2 hour taking this me

rtility

ldbearing potential control methods s after stopping both

chines

• During this 4-h fat-free (skimm or coffee can b use cream, wh cow’s milk, no or sweeteners. (skimmed) cow coffee.
• Two hours afte

ligible influence on use machines. You ackage leaflet of as that medicine may

da

resume norma

Figure 1. Dose t

cine exactly as your heck with your f you are not sure uld be used.

olda to take

stat) capsules must be glucosidase alfa. See also aflet of cipaglucosidase

0 kg or more, the

dose is 4 capsules

h 65 mg of miglustat.

etween 40 kg and 50 kg, ded dose is 3 capsules.

ke Opfolda

e Opfolda and

se alfa once every other e used on the same day. icines exactly as you

to by your doctor, see s so your treatment can s possible.

od

pfolda by mouth on an

s before and 2 hours after dicine.

our fasting period, water, ed) cow’s milk, and tea e consumed. Do not ole / semi-skimmed n-dairy milks, sugar, You can have fat-free ’s milk with your tea or

r taking Opfolda, you can

l eating and drinking.

imeline

Switc repla

If you anoth

• You oth

• Tell you

  hing from another enzyme cement therapy (ERT)

  are currently being treated with er ERT:

  r doctor will tell you when to stop the er ERT before starting Opfolda. your doctor when you completed r last dose.

If you shoul

Tell yo hospi capsu may b side e

take more Opfolda than you d ur doctor immediately or go to the tal if you accidentally take more les than you were prescribed. You e at increased risk of experiencing ffects with this medicine (see sectio appro

If you

If you speak your d resch cipag

n 4). Your doctor will provide priate supportive care.

forget to take Opfolda miss a dose of Opfolda, please to your doctor or nurse. Contact octor or nurse immediately to edule miglustat in combination with lucosidase alfa as soon as possible.

If you

Spea Opfol disea

4. Po

Like a

stop taking Opfolda k to your doctor if you wish to stop da treatment. The symptoms of your se may worsen if you stop treatment.

ssible side effects ll medicines, this medicine can cause ffects, although not everybody gets da is used with cipaglucosidase alfa, ide effects can occur with either of medicines.

llowing side effects may occur:

Allerg Allerg such puffy cough throat

ic reactions ic reactions may include symptoms as rash anywhere on the body, eyes, prolonged difficulty breathing, , swelling of the lip, tongue, or , itchy skin, and hives.

VV-LAB-003344 - V1.0

Tell a doctor or nurse immediately if yo are experiencing or think you may be experiencing allergic reactions. Inform doctor or nurse if you have ever had a such reaction.

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

u

your

ny

• Mouth pain
• Skin discolouration
• Pain in the area between the hi
• Muscle tiredness
• Muscle stiffness
• Weakness
• Pain in the cheek, gums, lips, c
• Feeling jittery
• Headache

Common (may affect up to 1 in 10 pe

• Serious life-threatening allergic react (anaphylactic reaction)
• Tremor
• Taste disturbance

• Sensation like numbness, tingling, p

  ople)

  ion

  ins,

  • Pain in chest

Reporting of side effects

If you get any side effects, talk to doctor or nurse. This includes an side effects not listed in this leafl can also report side effects direc details below). By reporting side

and needles (paraesthesia)

• Rapid heartbeat
• Low blood pressure
• Shortness of breath
• Diarrhoea
• Feeling sick (nausea)
• Stomach pain
• Passing gas
• Bloating
• Vomiting
• Constipation
• Hives
• Rash
• Itchy skin
• Excessive sweating
• Painful muscle contractions
• Muscle pain
• Muscle weakness
• Joint pains
• Tiredness
• Fever
• Chills
• Swelling in the hands, feet, ankles, l
• Rise in blood pressure

Uncommon (may affect up to 1 in 100

egs

date refers to the last date of tha

This medicine does not require a storage conditions.

Do not throw away any medicine wastewater or household waste. pharmacist how to throw away m

people)

• Allergic reaction
• Cannot hold or maintain balance
• Migraine
• Unusual paleness of the skin
• Asthma
• Uneasy stomach

• Painful contractions in gullet

  Capsule shell

  Gelatin

  Titanium dioxide (E171) Black iron oxide (E172)

  Edible printing ink

  Black iron oxide (E172) Potassium hydroxide (E525

5. How to store Opfolda

Keep this medicine out of the sig reach of children.

Do not use this medicine after th date which is stated on the bottle carton after the letters “EXP”. Th

ht and

e expiry and

e expiry

Propylene glycol (E1520) Strong ammonia solution (E Shellac (E904)

What Opfolda looks like a the pack

Bottles of 4 and 24 capsule

t month.

ny special

s via

Ask your edicines

Not all pack sizes may be m Size 2 hard capsule with a cap and white opaque bod printed in black on the bod white to off-white powder.

Marketing Authorisation H

Amicus Therapeutics UK Li One Globeside

that you no longer use. These m will help protect the environment

easures

. Fieldhouse Lane

Marlow, Buckinghamshire SL7 1HZ

nd other

iglustat. ns 65 mg of

Manufacturer

Manufacturing Packa B.V.

Neptunus 12, Heerenv Netherlands

This leaflet was last

:

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460i)

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527)

nd contents of

s.

arketed.

grey opaque y with “AT2221” y, containing

older

mited

ging Farmaca (MPF)

een, 8448CN,

revised in 11/2024

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p and rib

hin

6. Contents of the pack a information

What Opfolda contains

• The active substance is m Each hard capsule contai miglustat.

your

y possible et. You

tly (see

effects

• The other ingredients are

Capsule contents

Pregelatinised starch (maiz Magnesium stearate (E470b Microcrystalline cellulose (E Sucralose (E955)

Colloidal silicon dioxide

you can help provide more inform the safety of this medicine.

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