Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - palynziq

Document Subject

Generated Narrative: MedicinalProductDefinition mpc0c73074469385390d12a06f439c2f95

identifier: http://ema.europa.eu/identifier/EU/1/19/1362/001 1 x 2.5 mg pre-filled syringe

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Palynziq 2.5 mg solution for injection in pre-filled syringe

type: Full name

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part: nan

type: Invented name part

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part: nan

type: Scientific name part

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type: Strength part

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type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-c0c73074469385390d12a06f439c2f95

identifier: http://ema.europa.eu/identifier/EU/1/19/1362/001 1 x 2.5 mg pre-filled syringe

status: Final

type: Package Leaflet

category: Raw

date: 2022-02-16 13:28:17+0000

author: Organization ACME industry

title: TEST PURPOSES ONLY - palynziq

Attesters

What is in this leaflet

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

Palynziq contains the active substance pegvaliase, an enzyme that can break down a substance called phenylalanine in the body. Palynziq is a treatment for patients aged 16 years and older with phenylketonuria (PKU), a rare inherited disorder that causes phenylalanine from proteins in food to build up in the body. People who have PKU have high levels of phenylalanine and this can lead to serious health problems. Palynziq reduces the levels of phenylalanine in the blood of patients who have PKU whose blood phenylalanine levels cannot be kept below 600 micromol/l by other means such as by diet.

Do not use Palynziq

• if you have a severe allergy to pegvaliase or any other ingredients of this medicine, or another medicine that contains polyethylene glycol (PEG) (listed in section 6).

Warnings and precautions

Talk to your doctor, pharmacist, or nurse before using Palynziq.

Allergic reactions
You may have allergic reactions when being treated with Palynziq. Your doctor will tell you how to manage your allergic reactions based on the severity of the reaction, and will prescribe you additional medicines to manage the reaction.

Before using Palynziq, tell your doctor if you cannot use or do not want to use an adrenaline injection device to treat a severe allergic reaction to Palynziq.

Palynziq can cause severe allergic reactions that may be life-threatening and these can happen any time after a Palynziq injection.

• Stop injecting Palynziq if any of the following symptoms occur.

• Swelling of the face, eyes, lips, mouth, throat, tongue, hands and/or feet
• Trouble breathing or wheezing
• Throat tightness or choking feeling
• Trouble swallowing or speaking
• Feeling dizzy or fainting
• Losing control of urine or stools
• Rapid heartbeat
• Hives (like an itchy, bumpy skin rash) that spreads quickly
• Flushing
• Severe stomach cramps or pain, vomiting, or diarrhoea

• Use adrenaline injection device as instructed by your doctor and seek urgent medical assistance.

Your doctor will prescribe an adrenaline injection device to use for a severe allergic reaction. Your doctor will train you and someone helping you on when and how to use adrenaline. Keep the adrenaline injection device with you at all times.

For at least the first 6 months of treatment, someone must be with you when you are self-injecting Palynziq. This person must stay with you for at least 1 hour after your injection to watch you for signs and symptoms of a severe allergic reaction and, if needed, give you an injection of adrenaline and call for emergency medical help.

If you have a severe allergic reaction, do not continue to use Palynziq until you have talked with the doctor who prescribes you Palynziq. Tell your doctor that you had a severe allergic reaction. Your doctor will tell you if you can continue Palynziq treatment.

Time needed to lower your blood phenylalanine levels Your doctor will start you on Palynziq at a low dose and will increase your dose slowly. It will take time to find the dose that works best to lower your blood phenylalanine levels. Most people respond within 18 months, but it can sometimes take up to 30 months.

Injection of other medicines that contain PEG while using Palynziq
Palynziq includes an ingredient called polyethylene glycol (PEG). If you inject Palynziq with another injectable medicine that contains PEG, such as PEGylated medroxyprogesterone acetate, you may have an allergic reaction. Tell your doctor or pharmacist if you are injecting, have recently injected or might inject any other medicines.

Blood phenylalanine levels that are too low You may have blood phenylalanine levels that are too low when using Palynziq. Your doctor will check your blood phenylalanine levels monthly. If your blood phenylalanine levels are too low, your doctor may ask you to change your diet and/or will lower your dose of Palynziq. Your doctor will check your blood phenylalanine levels every 2 weeks until your blood phenylalanine levels return to normal.

Children and adolescents It is not known if Palynziq is safe and effective in children and adolescents less than 16 years of age with PKU and therefore should not be used in people aged less than 16 years.

Other medicines and Palynziq Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines.

Pregnancy, breast-feeding and fertility 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 using this medicine.

Palynziq is not recommended during pregnancy unless your condition requires treatment with Palynziq and other ways of controlling your blood phenylalanine levels do not work. If your phenylalanine levels are too high or too low during pregnancy, this may harm you or your baby. You and your doctor will decide the best way for you to manage your blood phenylalanine levels. It is very important to keep your phenylalanine levels under control before and during pregnancy.

It is not known if Palynziq passes into breast milk or if it will affect your baby. Talk to your healthcare provider about the best way to feed your baby if you use Palynziq.

It is not known if Palynziq has an effect on fertility. Animal studies suggest that women may have difficulty becoming pregnant if their phenylalanine levels are abnormally low.

Driving and using machines Palynziq can affect your ability to drive and use machines if you have a severe allergic reaction.

Palynziq contains sodium
This medicine contains less than 1 mmol sodium (23 mg) per pre-filled syringe, that is to say essentially sodium-free .

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

Palynziq is given as an injection under the skin (subcutaneous injection).

Dose

• You will start Palynziq at the lowest dose. You will use the 2.5 mg syringe once a week for at least the first 4 weeks. The 2.5 mg syringe has a white plunger.
• Your doctor will then slowly increase your dose and/or how often you inject Palynziq. Your doctor will tell you how long to stay at each dose. Slowly increasing your dose over time allows your body to adjust to this medicine.
• The goal is to reach a daily dose that lowers your blood phenylalanine levels to within the target range of 120 to 600 micromol/l and does not cause too many side effects. Patients usually need a daily dose of 20 mg, 40 mg, or 60 mg to reach their target blood phenylalanine level.

Example of steps to reach your blood phenylalanine goal

Palynziq dose and how often to take it Syringe colour

2.5 mg once a week White plunger 2.5 mg two times a week 10 mg once a week Green plunger

10 mg twice a week 10 mg four times a week 10 mg daily 20 mg daily Blue plunger

40 mg daily (2 injections of 20 mg pre-filled syringe)1 60 mg daily (3 injections of 20 mg pre-filled syringe)1 1 If you need more than one injection to receive your daily dose, all injections should be done at the same time of day and injection sites should be at least 5 centimetres away from each other. Do not divide your daily dose throughout the day.

• Your doctor will continue to check your blood phenylalanine levels during treatment and may adjust your dose of Palynziq or ask you to change your diet.
• Your doctor will need to check your blood phenylalanine monthly to see if this medicine is working for you.

Starting Palynziq

• Your healthcare provider will give you the Palynziq injection until you (or a caregiver) can do it yourself.
• Your doctor will prescribe medicines for you to take before your Palynziq injection, such as paracetamol, fexofenadine, and/or ranitidine. These medicines help to reduce the symptoms of an allergic reaction.
• A healthcare provider will monitor you for at least 1 hour after you get Palynziq for signs and symptoms of an allergic reaction.
• Your doctor will also prescribe adrenaline injection device to use for any severe allergic reactions. Your healthcare provider will also tell you which signs and symptoms to look out for and what to do if you have a severe allergic reaction.
• Your doctor will show you how and when to use the adrenaline injection device. Keep it with you at all times.

Continuing Palynziq

• This medicine comes in pre-filled syringes with 3 different strengths (2.5 mg-white plunger, 10 mg-green plunger, or 20 mg-blue plunger). You may need more than one pre-filled syringe for your prescribed dose. Your healthcare provider will tell you which syringe, or a combination of syringes, to use and will show you (or a caregiver) how to inject Palynziq.
• The Instructions for Use (section 7 of this leaflet) shows you: * how to prepare and inject Palynziq and * how to throw away Palynziq syringes properly after you use them
• Your doctor will tell you how long to continue taking medicines such as paracetamol, fexofenadine, and/or ranitidine before you take Palynziq.
• For at least the first 6 months of Palynziq treatment, you must have someone with you when you self-inject Palynziq, and for at least 1 hour after your injection to watch for signs and symptoms of a severe allergic reaction and, if needed, give you an injection of adrenaline and call for emergency medical help.

Your doctor will train them on the signs and symptoms of a severe allergic reaction and how to give an injection of adrenaline. * Your doctor will tell you if you need an observer for longer than 6 months.

• Do not change your protein intake unless your doctor tells you to.

If you use more Palynziq than you should If you use more Palynziq than you should, tell your doctor. See section 4 for details on what to do based on your symptoms.

If you forget to use Palynziq If you miss a dose, take your next dose at the regular time. Do not take two doses of Palynziq to make up for a missed dose.

If you stop taking Palynziq If you stop taking Palynziq treatment, your blood phenylalanine levels are likely to increase. Talk to your doctor before stopping Palynziq treatment.

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

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

Allergic reactions occur very commonly (may affect more than 1 in 10 people) and range in severity. Symptoms of allergic reaction can include skin rash, itching, swelling of head or face, itchy or runny eyes, cough, trouble breathing, wheezing, and feeling dizzy. Your doctor will tell you how to manage any allergic reactions based on their severity, and will prescribe you additional medicines to manage the reaction. Some of these allergic reactions can be more serious, as described below, and will require immediate attention.

Serious side effects include:

• Sudden severe allergic reactions: (Common may affect up to 1 in 10 people). Stop injecting Palynziq if you notice any serious sudden signs of allergy or combination of signs listed below. * Swelling of the face, eyes, lips, mouth, throat, tongue, hands and/or feet * Trouble breathing or wheezing * Throat tightness or a choking feeling * Trouble swallowing or speaking * Feeling dizzy or fainting * Losing control of urine or stools * Rapid heartbeat * Hives (like an itchy, bumpy skin rash) that spreads quickly * Flushing * Severe stomach cramps or pain, vomiting, or diarrhoea

Use adrenaline injection device as instructed by your doctor and seek urgent medical assistance. Your doctor will prescribe an adrenaline injection device to use for a severe allergic reaction. Your doctor will train and instruct you and someone helping you on when and how to use adrenaline. Keep the adrenaline injection device with you at all times.

Contact your doctor immediately if you have the following:

• A type of allergic reaction called serum sickness which includes a combination of fever (high temperature), rash, muscle and joint aches (Common may affect up to 1 in 10 people)

Other side effects

Very common: may affect more than 1 in 10 people

• skin redness, swelling, bruising, tenderness, or pain where you injected Palynziq
• joint pain
• decrease in complement factors C3 and C4 proteins (which are parts of your immune system) in blood test
• allergic reaction
• too low levels of phenylalanine in blood tests
• headache
• skin rash
• stomach pain
• feeling sick, also called nausea
• vomiting
• hives (raised itchy rash on the skin)
• itchiness
• thinning or loss of hair
• cough
• increase in c-reactive protein (CRP) in blood test (CRP is a protein that indicates that you have inflammation)
• swollen glands in the neck, armpit or groin
• skin redness
• muscle pain
• diarrhoea
• fatigue (feeling tired)

Common: may affect up to 1 in 10 people

• trouble breathing
• joint stiffness
• joint swelling
• muscle stiffness
• skin rash with small bumps
• blistering or peeling of the outer layer of the skin

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 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.

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 syringe label, tray cover, and carton after EXP . The expiry date refers to the last day of that month.

Store in a refrigerator (2 C 8 C). Do not freeze.

If needed, you may store Palynziq in its sealed tray outside the refrigerator (below 25 C) for up to a single period of 30 days away from sources of heat. Record the date removed from refrigeration on the unopened product tray. Once stored outside of refrigeration, the product must not be returned to the refrigerator.

Do not use this medicine if the pre-filled syringe is damaged or you notice the solution is discoloured, cloudy, or if you can see particles.

Use safe disposal procedures for syringes. 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 will help protect the environment.

What Palynziq contains

• The active substance is pegvaliase. Each 2.5 mg pre-filled syringe contains 2.5 mg pegvaliase in 0.5 ml solution. Each 10 mg pre-filled syringe contains 10 mg pegvaliase in 0.5 ml solution. Each 20 mg pre-filled syringe contains 20 mg pegvaliase in 1 ml solution.
• The other ingredients are trometamol, trometamol hydrochloride, sodium chloride (see section 2 for further information), trans-cinnamic acid, water for injections.

What Palynziq looks like and contents of the pack Palynziq solution for injection (injection) is a clear to slightly opalescent, colourless to pale yellow solution. The pre-filled syringe includes an automatic needle guard.

Pre-filled syringe 2.5 mg (white plunger): Each 2.5 mg carton contains 1 pre-filled syringe.

Pre-filled syringe 10 mg (green plunger): Each 10 mg carton contains 1 pre-filled syringe.

Pre-filled syringe 20 mg (blue plunger): Each 20 mg carton contains 1 or 10 pre-filled syringes.

Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer BioMarin International Limited Shanbally, Ringaskiddy
County Cork
Ireland P43 RThis leaflet was last revised in MM/YYYY.

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site: