Bylvay

Package leaflet: Information for the patient

Bylvay 200 microgram capsules, 400 microgram capsules, 600 microgram capsules, 1200 microgram capsules

odevixibat
This medicinal product is subject to additional monitoring. This will allow for rapid identification of new safety information. You can help by reporting any side effects you experience while taking this medicine (see the end of section 4 for information on how to report side effects).
Please read this leaflet carefully before taking this medicine, as it contains important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, consult your doctor or pharmacist.
• This medicine has been prescribed for you only. Do not give it to other people, even if their symptoms are the same as yours, as it may be harmful.
• If you experience any side effect, including those not listed in this leaflet, consult your doctor or pharmacist. See section 4.

Contents of this leaflet

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

1. What Bylvay is and what it is used for

Bylvay contains the active substance odevixibat. Odevixibat is a medicine that increases the removal of substances called bile acids from the body. Bile acids are components of a digestive fluid called bile, which is produced by the liver and secreted into the intestine. Odevixibat blocks the mechanism that normally reabsorbs them from the intestine after they have performed their function. This allows them to be excreted from the body in the faeces.
Bylvay is used to treat progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months and older. PFIC is a liver disease caused by the accumulation of bile acids (cholestasis) that worsens over time and is often accompanied by severe itching.

2. What you need to know before taking Bylvay

Do not take Bylvay

• if you are allergic to odevixibat or to 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 complete absence or lack of function of the bile acid efflux pump protein;
• you have severely reduced liver function;
• you have reduced stomach or intestinal motility, or reduced circulation of bile acids between the liver, bile, and small intestine due to medications, surgical procedures, or diseases other than PFIC. These conditions may reduce the effect of odevixibat.

If you develop diarrhea while taking Bylvay, consult your doctor. Patients experiencing diarrhea are advised to drink sufficient fluids to prevent dehydration.
During treatment with Bylvay, an increase in liver enzyme levels may be observed in liver function tests. Before starting Bylvay, your doctor will measure your liver function to ensure your liver is working properly. Your doctor will perform regular monitoring of your liver function.
Before and during treatment, your doctor may also check your blood levels of vitamin A, D, and E, as well as your INR (International Normalized Ratio, which measures bleeding risk).

Children
Bylvay is not recommended for children under 6 months of age, as it is not known whether the medicine is safe and effective in this age group.

Other medicines and Bylvay
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Treatment with odevixibat may affect the absorption of fat-soluble vitamins such as vitamins A, D, and E, and other fat-soluble medicines.

Pregnancy and breastfeeding
If you are pregnant, think you might be pregnant, are planning to become pregnant, or are breastfeeding, consult your doctor before taking this medicine.
Bylvay is not recommended during pregnancy and in women of childbearing potential who are not using contraceptive measures.
It is not known whether odevixibat passes into breast milk and could affect the breastfed infant. Your doctor will help you decide whether to discontinue breastfeeding or avoid treatment with Bylvay, taking into account the benefits of breastfeeding for the infant and the benefits of Bylvay for the mother.

Driving and using machines
Bylvay does not affect or affects negligibly the ability to drive and use machines.

3. How to take Bylvay

Take this medicine exactly as your doctor or pharmacist has told you. If
you have any doubts, consult your doctor or pharmacist.
The dose of Bylvay depends on body weight. Your doctor will determine the correct number and strength of capsules to take.
The recommended dose is

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

No dose adjustment is recommended for adults.
Method of administration
Take the capsules once daily in the morning, with or without food.
All capsules may be swallowed whole with a glass of water or opened and sprinkled onto food or dispersed in a liquid suitable for the patient's age (e.g., breast milk, infant formula, or water).
The larger 200- and 600-microgram capsules are intended to be opened and sprinkled onto food or dispersed in an age-appropriate liquid, but may also be swallowed whole.
The smaller 400- and 1,200-microgram capsules are intended to be swallowed whole, but may be opened and sprinkled onto food or dispersed in an age-appropriate liquid.
Detailed instructions for opening the capsules and sprinkling the contents onto food or into a liquid are provided at the end of this leaflet.
If the medicine does not improve your condition after 6 months of continuous daily treatment, your doctor will advise you on an alternative treatment.
If you take more Bylvay than you should
Inform your doctor if you think you have taken too much Bylvay.
Possible symptoms of overdose include diarrhoea and stomach or intestinal problems.
If you forget to take Bylvay
Do not take a double dose to make up for the missed dose. Take the next dose at your usual time.
If you stop taking Bylvay
Do not stop treatment with Bylvay without first consulting your doctor.
If you have any questions about how to use 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.
The following side effects may occur with the listed frequency:

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

• diarrhoea, including diarrhoea with blood in the stool, soft stools
• vomiting
• abdominal pain (stomach ache)
• increased bilirubin (a substance produced by the liver) measured in blood
• increased levels of liver enzymes (ALT) measured in blood
• low levels of vitamin D in the blood

Common (may affect up to 1 in 10 people)

• enlarged liver
• increased levels of liver enzymes (AST) measured in blood
• low levels of vitamin E in the blood

If laboratory test results are abnormal, ask your doctor what this might mean.

Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, please inform your doctor or pharmacist. 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 Bylvay

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the carton and vial after
Exp. The expiry date refers to the last day of that month.
Store in the original packaging to protect the medicine from light. Do not store above 25 °C.
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 Bylvay contains

• The active substance is odevixibat. Each Bylvay 200 microgram hard capsule contains 200 micrograms of odevixibat (as sesquihydrate). Each Bylvay 400 microgram hard capsule contains 400 micrograms of odevixibat (as sesquihydrate). Each Bylvay 600 microgram hard capsule contains 600 micrograms of odevixibat (as sesquihydrate). Each Bylvay 1,200 microgram hard capsule contains 1,200 micrograms of odevixibat (as sesquihydrate).

The other components are:

• Capsule contents:
  Microcrystalline cellulose
  Hypromellose

Capsule shell
Bylvay 200 micrograms and 600 micrograms hard capsules
Hypromellose
Titanium dioxide (E171)
Yellow iron oxide (E172)
Bylvay 400 micrograms and 1,200 micrograms hard capsules
Hypromellose
Titanium dioxide (E171)
Yellow iron oxide (E172)
Red iron oxide (E172)
Printing ink
Shellac
Propylene glycol (E1520)
Black iron oxide (E172)

Description of the appearance of Bylvay and contents of the pack
Bylvay 200 microgram hard capsules:
Size 0 (21.7 mm × 7.64 mm) capsules with an opaque ivory cap and an opaque white body, printed with "A200" in black ink.
Bylvay 400 microgram hard capsules:
Size 3 (15.9 mm × 5.82 mm) capsules with an opaque orange cap and an opaque white body, printed with "A400" in black ink.
Bylvay 600 microgram hard capsules:
Size 0 (21.7 mm × 7.64 mm) capsules with an opaque ivory cap and body, printed with "A600" in black ink.
Bylvay 1,200 microgram hard capsules:
Size 3 (15.9 mm × 5.82 mm) capsules with an opaque orange cap and body, printed with "A1200" in black ink.

Bylvay hard capsules are packed in a plastic bottle with a tamper-evident and child-resistant closure. Pack: 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 further information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

België/Belgique/Belgien/Luxembourg/Luxemburg
Ipsen NV België/Belgique/Belgien
Tel: +32 9 243 96 00

Italia
Ipsen SpA
Tel: +39 02 39 22 41

България
Swixx Biopharma EOOD
Tel: +359 (0)2 4942 480

Latvija
Ipsen Pharma representative office
Tel: +371 67622233

Česká republika
Ipsen Pharma s.r.o
Tel: +420 242 481 821

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 5930

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

France
Ipsen Pharma
Tél: +33 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 medicine has been authorised under “exceptional circumstances”. This means that, because the disease is rare, it has not been possible to obtain complete information about this medicine. The European Medicines Agency will review any new information on this medicine annually, and this package leaflet will be updated if necessary.

Other sources of information
More detailed information on this medicine is available on the European Medicines Agency website: https://www.ema.europa.eu.
In addition, links to other websites on rare diseases and their therapeutic treatments are provided.

Instructions
Instructions for opening the capsules and sprinkling the contents onto food:
Step 1. Place a small amount of soft food in a bowl (2 tablespoons/30 mL of yoghurt, apple puree, banana or carrot puree, chocolate pudding, rice pudding, or oat porridge). The food should be at room temperature or cooler.

	Step 2: •Hold the capsule horizontally at both ends and twist in opposite directions.
	Step 3: •Separate the capsule halves to empty the contents into the bowl with soft food. •Gently tap the capsule to ensure all granules are released. •Repeat the previous step if the prescribed dose requires more than one capsule.
	Step 4: •Gently mix the capsule contents into the soft food.
Take the entire dose immediately after mixing. Do not store the mixture for later use. •Drink a glass of water after taking the dose. •Dispose of the empty capsule shells.

Instructions for opening the capsules and pouring the contents into a liquid suitable for the patient's age:
Do not administer using a bottle or a sippy cup, as the granules cannot pass through the opening. The granules do not dissolve in the liquid.
Contact the pharmacy if an appropriate oral administration syringe is not available at home.

	Step 1: • Hold the capsule horizontally at both ends and twist them in opposite directions. • Separate the two parts and empty the contents into a dosing cup or a small cup. • Gently tap the capsule to ensure all granules are released. Repeat this step if the prescribed dose requires more than one capsule.
	Add 1 teaspoon (5 mL) of a liquid appropriate for the child's age (e.g., breast milk, infant formula, or water). • Let the granules sit in the liquid for about 5 minutes to ensure they are fully wetted (the granules do not dissolve).
	Step 2: • After 5 minutes, place the tip of the oral syringe completely into the dosing cup. • Slowly pull back the plunger to draw the liquid/granule mixture into the syringe. Gently push the plunger and expel the liquid/granule mixture back into the dosing cup. Repeat this process 2 or 3 times to ensure the granules are thoroughly mixed with the liquid.
	Step 3: • Draw the entire contents into the oral syringe by pulling back the plunger to the end of the syringe.
	Step 4: • Place the tip of the oral syringe into the front part of the child’s mouth, between the tongue and the inside of the cheek. Gently push the plunger to release the liquid/granule mixture between the tongue and the inside of the child’s cheek. Do not administer the liquid/granule mixture at the back of the child’s throat, as this may cause gagging or choking.

• If any of the liquid/granule mixture remains in the dosing cup, repeat steps 3 and 4 until the entire dose has been administered. Administer the complete dose immediately after mixing. Do not store the liquid/granule mixture for later use.
• After administering the dose, give breast milk, infant formula, or another age-appropriate liquid.
• Dispose of all empty capsule sachets.