InfoCons Consumer Protection informs you – Bylvay – Package Leaflet

Before anything else

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

• Keep this leaflet. You may need to read it again.
• If you have any further questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet . See section 4.

ℹ️ 1. What Bylvay is and what it is used for

Bylvay contains the active substance odevixibat. Odevixibat is a medicine which increases the removal of substances called bile acids from the body. Bile acids are components of the digestive fluid called bile, which is produced by the liver and secreted into the intestines. Odevixibat blocks the mechanism that normally reabsorbs them from the intestines after they have done their job. This allows them to pass out of the body in the stool .

Bylvay is used to treat progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. PFIC is a liver disease caused by build -up of bile acids (cholestasis ) that gets worse over time and is often accompanied with severe itch ing.

⚠️ 2. What you need to know before you take Bylvay

Do not take Bylvay

• if you are allergic to odevixibat or any of the other ingredients of this medicine (listed in section 6)

Warnings and precautions

Talk to your doctor or pharmacist before taking Bylvay if you have:

• been diagnosed with a complete absence or lack of function of bile salt export pump protein
• severely reduced liver function
• reduced stomach or bowel motility, or reduced circulation of bile acids between liver, bile and small intestine due to medicines , surgical procedures or diseases other than PFIC 40 since these may reduce the effect of odevixibat Talk to your doctor if you develop diarrhoea while taking Bylvay. Drinking sufficient liquid is recommended in patients with diarrhoea to prevent dehydration .

Increased levels in liver enzymes might be seen in liver function tests when taking Bylvay. Before you start taking Bylvay, your doctor will measure your liver function to check how well your liver is working. Your doctor will do regular checks to monitor your liver function.

Before and during treatment, your doctor may also check your blood levels of vitamin A, D and E and your INR (international normalised ratio, which measures your risk for bleeding) .

Children

Bylvay is not recommended for babies under 6 months because it is not known if the medicine is safe and effective in this age group.

Other medicines and Bylvay

Tell your doctor or pharmacist if you are using, have recently used or might use any other medicines .

Treatment with odevixibat may affect the absorption of fat -soluble vitamins such as vitamin A, D and E, and other fat-soluble medicines .

Pregnancy and breast-feeding

If you are pregnant or breast -feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine .

Bylvay is not recommended during pregnancy and in women of childbearing potential not using contraception.

It is not known if odevixibat can pass into breast milk and affect the baby. Your doctor will help you to decide whether to stop breast-feeding or avoid Bylvay treatment, considering the benefit of breast – feeding to the baby and Bylvay to the mother .

Driving and using machines

Bylvay has no or negligible influence on the ability to drive or capacity to use machinery.

💊 3. How to take Bylvay

Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor or pharmacist if you are not sure.

The dose of Bylvay is based on your weight. Your doctor will work out the right number and strength of capsules for you to take.

The recommended dose is

• 40 micrograms odevixibat per kilogram body weight once daily
• If the medicine is not working well enough after 3 months, your doctor may increase the dose to 120 micrograms odevixibat per kilogram body weight (up to a m aximum of 7 200 micrograms once daily ).

No dose differences are recommended for adults .

41 Method of use Take the capsules once daily in the morning with or without food .

All capsules can be either swallowed whole with a glass of water or opened and sprinkled on food or in an age-appropriate liquid (e.g. breast milk, infant formula or water) .

The larger 200 and 600 micrograms capsules are intended to be opened and sprinkled on food or in an age-appropriate liquid but may be swallowed whole.

The smaller 400 micrograms and 1 200 micrograms capsules are intended to be swallowed whole but may be opened and sprinkled on food or in an age -appropriate liquid .

Detailed instructions on how to open capsules and sprinkle on food or in a liquid can be found at the end of this package leaflet.

If the medicine does not improve your condition after 6 months of continuous daily treatment , your doctor will recommend an alternative treatment.

If you take more Bylvay than you should

Tell your doctor if you think you have taken too much Bylvay .

Possible overdose symptoms are diarrhoea , stomach and bowel problems.

If you forget to take Bylvay

Do not take a double dose to make up for a forgotten dose. Take the next dose at the usual time.

If you stop taking Bylvay

Do not stop taking Bylvay without first discussing with your doctor.

If you have any further questions on the use of this medicine, ask your doctor or pharmacist .

❗ 4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Side effects may occur with the following frequenc y:

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

• diarrhoea, including diarrhoea with bloody stool, soft stools
• vomiting
• abdominal (belly) pain
• increase in bilirubin (a substance produced by the liver ) as measured in the blood
• increase in liver enzyme (ALT) as measured in the blood
• low vitamin D blood level

Common (may affect up to 1 in 10 people)

• enlarged liver
• increase in liver enzyme (AST) as measured in the blood
• low vitamin E blood level If you have abnormal lab oratory values, talk to your doctor about what this means to you.

Reporting of side effects

If you get any side effects , talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V . By reporting side effects , you can help provide more information on the safety of this medicine.

🌡️ 5. How to store Bylvay

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 carton and bottle after EXP . The expiry date refers to the last day of that month.

Store in the original package to protect from light . Do not store above 25 °C.

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures wil l help protect the environment.

📦 6. Contents of the pack and other information

What Bylvay contains

• The active substance is odevixibat.

Each Bylvay 200 micrograms hard capsule contains 200 micrograms odevixibat (as sesquihydrate).

Each Bylvay 4 00 micrograms hard capsule contains 400 micrograms odevixibat (as sesquihydrate).

Each Bylvay 6 00 micrograms hard capsule contains 600 micrograms odevixibat (as sesquihydrate).

Each Bylvay 1 200 micrograms hard capsule contains 1 200 micrograms odevixibat (as sesquihydrate).

• The other ingredients are :

Capsule content Microcrystalline cellulose Hypromellose Capsule shell Bylvay 200 microgram s and 600 microgram s hard capsules Hypromellose Titanium dioxide (E171) Yellow iron oxide (E172) Bylvay 400 microgram s and 1 200 microgram s hard capsules Hypromellose Titanium dioxide (E171) Yellow iron oxide (E172) Red iron oxide (E172) Printing ink Shellac 43 Propylene glycol (E1520) Black iron oxide (E172)

What Bylvay looks like and contents of the pack

Bylvay 200 micrograms hard capsules :

Size 0 capsules (21.7 mm × 7.64 mm) with ivory opaque cap and white opaque body; imprinted “A200” with black ink.

Bylvay 400 micrograms hard capsules :

Size 3 capsules (15.9 mm × 5.82 mm) with orange opaque cap and white opaque body; imprinted “A400” with black ink.

Bylvay 600 micrograms hard capsules:

Size 0 capsules (21.7 mm × 7.64 mm) with ivory opaque cap and body; imprinted “A600” with black ink.

Bylvay 1 200 micrograms hard capsules:

Size 3 capsules (15.9 mm × 5.82 mm) with orange opaque cap and body; imprinted “A1200” with black ink.

Bylvay hard capsules are packed in a plastic bottle with a tamper evident, child resistant polypropylene closure. Pack size: 30 hard capsules.

Marketing Authorisation Holder

Ipsen Pharma 70 rue Balard 75015 Paris France

Manufacturer

Almac Pharma Services Limited Seagoe Industrial Estate Portadown, Craigavon County Armagh BT63 5UA United Kingdom (Northern Ireland) For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

België/Belgique/Belgien/Luxembourg/ Luxemburg Ipsen NV België/Belgique/Belgien Tél/Tel: + 32 9 243 96 00 Italia Ipsen SpA Tel: + 39 02 39 22 41 България Swixx Biopharma EOOD Teл.: +359 (0)2 4942 480 Latvija Ipsen Pharma representative office Tel: + 371 67622233 44 Česká republika Ipsen Pharma s.r.o Tel: +     Lietuva Ipsen Pharma SAS Lietuvos filialas Tel: +370 700 33305 Danmark , Norge, Suomi/Finland, Sverige, Ísland Institut Produits Synthèse (IPSEN) AB Sverige/Ruotsi/Svíþjóð Tlf/Puh/Tel/Sími: +46 8 451 60 00 Magyarország IPSEN Pharma Hungary Kft.

Tel.: + 36 1 555 593 0 Deutschland , Österreich Ipsen Pharma GmbH Deutschland Tel: +49 89 2620 432 89 Nederland Ipsen Farmaceutica B.V. Tel: +31 (0) 23 554 1600 Eesti Centralpharma Communications OÜ Tel: +372 60 15 540 Polska Ipsen Poland Sp. z o.o.

Tel.: + 48 22 653 68 00 Ελλάδα, Κύπρος, Malta Ipsen Μονοπρόσωπη EΠΕ Ελλάδα Τηλ: +30 210 984 3324 Portugal Ipsen Portugal – Produtos Farmacêuticos S.A.

Tel: + 351 21 412 3550 España Ipsen Pharma, S.A.U.

Tel: +34 936 858 100 România Ipsen Pharma România SRL Tel: + 40 21 231 27 20 France Ipsen Pharma Tél : +33 (0)1 58 33 50 00 Slovenija Swixx Biopharma d.o.o.

Tel: + 386 1 2355 100 Hrvatska Swixx Biopharma d.o.o.

Tel: +385 1 2078 500 Slovenská republika Ipsen Pharma, organizačná zložka Tel: + 420 242 481 821 Ireland Ipsen Pharmaceuticals Limited Tel: +353 1 8098256

This leaflet was last revised in

This medicine has been authorised under ‘exceptional circumstances’. This means that because of the rarity of this disease it has been impossible to get complete information on this medicine.

The European Medicines Agency will review any new information on this medicine every year and this leaflet will be updated as necessary.

Other sources of information Detailed information on this medicine is available on the European Medicines Agency web site: https://www.ema.europa.eu .

There are also links to other websites about rare diseases and treatments.

45 Instructions Instructions to open capsules and sprinkle the contents on food:

Step 1. Place a small amount of soft food into a bowl (2 tablespoons/30 mL of yoghurt, apple sauce, banana or carrot puree, chocolate pudding, rice pudding or oatmeal porridge). Food should be at or below room temperature.

Step 2:

• Hold the capsule horizontally at both ends, twist in opposite directions.

Step 3:

• Pull apart to empty the contents into the bowl of soft food.
• Gently tap the capsule to ensure that all pellets come out
• Repeat the previous step if the dose requires more than one capsule.

Step 4:

• Gently mix the contents of the capsule into the soft food.
• Take the entire dose immediately after mixing. Do not store the mixture for future use.
• Drink a glass of water following the dose .
• Dispose of the empty capsule shells .

46 Instructions to open capsules and sprinkle the contents in an age-appropriate liquid :

Do not administer via a bottle or “sippy cup” because the pellets will not pass through the opening.

Pellets will not dissolve in liquids.

Contact your pharmacy if you do not have a suitable oral syringe for administration at home.

Step 1:

• Hold the capsule horizontally at both ends, twist in opposite directions.
• Pull apart and empty the contents into a small cup or glass.
• Gently tap the capsule to ensure that all pellets come out .

Repeat this if the dose requires more than one capsule .

• Add 1 teaspoon (5 ml) of an age-appropriate liquid (e.g. breast milk, infant formula or water).
• Let the pellets sit in the liquid for approximately 5 minutes to allow complete wetting (pellets will not dissolve).

Step 2:

• After 5 minutes, place the tip of the oral syringe completely into the mixing cup.
• Pull the plunger of the syringe up slowly to withdraw the liquid/pellet mixture into the syringe. Gently push the plunger down again to expel the liquid/pellet mixture back into the mixing cup. Do this 2 to 3 times to ensure complete mixing of the pellets into the liquid.

Step 3:

• Withdraw the entire contents into the oral syringe by pulling the plunger on the end of the syringe.

Step 4:

• Place the tip of the oral syringe into the front of the child’s mouth between the tongue and the side of the mouth, and then gently push the plunger down to squirt the liquid/pellet mixture between your child’s tongue and the side of the mouth. Do not squirt liquid/pellet mixture in the back of the child’s throat because this could cause gagging or choking.
• If any pellet/liquid mixture remains in the mixing cup, repeat Step 3 and Step 4 until the entire dose has been administered.
• Give the entire dose immediately after mixing. Do not store the liquid/pellet mixture for future use.

47 • Give breast milk, infant formula or other age -appropriate liquid to drink following the dose.

• Dispose of the empty capsule shells.

📄 Package Leaflet – official source

This article reproduces the information in the official package leaflet, as authorised in the European Union. You can consult the original document here: Product information Bylvay (PDF, ec.europa.eu).

You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/21/1566).

⚠️ Important: This article is for information purposes only and does not replace medical advice or the package leaflet inside the medicine box. Read the leaflet carefully before use. For any questions about your treatment, talk to your doctor or pharmacist. If you experience side effects, tell your doctor or pharmacist and report them through your national reporting system.