2.5 mg / 10 mg / 20 mg solution for injection in pre-filled syringe
pegvalias (pegvaliasum)
What Palynziq is and what it is used for
Palynziq contains the active substance pegvalias, 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 disease that causes phenylalanine from proteins in the diet to accumulate in the body. People with PKU have high levels of phenylalanine, which can lead to serious health problems. Palynziq lowers the levels of phenylalanine in the blood of patients with PKU whose phenylalanine levels in the blood cannot be kept below 600 micromol / l by other methods, for example by diet.
What you need to know before using Palynziq
Do not use Palynziq:
- if you have a severe allergy to pegvalias or any of the other ingredients of this medicine, or any other medicine containing polyethylene glycol (PEG) (listed in section 6).
Warnings and cautions
Talk to your doctor, pharmacist, or nurse before using Palynziq.
Allergic reactions
You may get allergic reactions when you are treated with Palynziq. Your doctor can tell you how to treat allergic reactions depending on the severity of the reaction and prescribe additional medicines to counteract the reaction.
Before you use Palynziq, tell your doctor if you can or do not want to use adrenaline injection equipment to treat a severe allergic reaction to Palynziq.
Palynziq can cause severe allergic reactions that can be life-threatening and these can occur at any time after injection of Palynziq.
- Stop injecting Palynziq if you get any of the following symptoms:
- swelling of the face, eyes, lips, mouth, throat, tongue, hands, and / or feet
- difficulty breathing or wheezing
- constriction in the throat or feeling of suffocation
- difficulty swallowing or speaking
- dizziness or fainting
- urinary or fecal incontinence
- fast heartbeat
- hives (itchy rash on the skin) that spreads rapidly
- redness
- severe abdominal pain or stomach cramps, vomiting, or diarrhea.
- Use the adrenaline injection equipment according to your doctor’s instructions and seek emergency care.
Your doctor will prescribe adrenaline injection equipment for you to use if you have a severe allergic reaction. The doctor trains you and someone to help you when and how to use adrenaline. Always carry the adrenaline injection kit.
During the first 6 months of treatment, someone must be with you when you inject Palynziq yourself.
The person must be with you for at least 1 hour after injection one and observe if you get signs and symptoms of a severe allergic reaction and, if necessary, give you an adrenaline injection and contact emergency care.
If you get a severe allergic reaction, do not continue to use Palynziq until you have talked to your doctor who has prescribed Palynziq for you. Tell your doctor that you have had a severe allergic reaction. Your doctor will tell you if you can continue to use Palynziq.
Time before the phenylalanine level in the blood is lowered
Your doctor will start by giving you a low dose of Palynziq and increase the dose slowly. It takes time to find the dose that works best to lower the phenylalanine levels in your blood. Most people respond to the medicine within 18 months, but it can sometimes take 30 months.
Injection of other medicines containing PEG while using Palynziq
Palynziq contains the ingredient polyethylene glycol (PEG). If you inject Palynziq while taking another injectable medicine containing PEG, such as pegylated medroxyprogesterone acetate, you may have an allergic reaction. Tell your doctor or pharmacist if you are injecting, have recently taken, or might inject other medicines.
Too low phenylalanine level in the blood
When you use Palynziq your blood phenylalanine levels may be too low. Your doctor checks the phenylalanine level in your blood every month. If you have too low a level of phenylalanine in your blood, your doctor may ask you to change your diet and/or reduce the dose of Palynziq you are receiving. Your doctor will check the phenylalanine level in your blood every two weeks until the phenylalanine level in your blood has returned to normal.
Children and young people
It is not known if Palynziq is safe and effective for children and adolescents under 16 years of age who have PKU, and therefore it should not be given to people under 16 years of age.
Other drugs and Palynziq
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.
Pregnancy, breastfeeding, and fertility
If you are pregnant or breastfeeding, 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 means of controlling phenylalanine levels do not work. If your phenylalanine level is too high or too low during pregnancy, it may harm you or your baby. You and your doctor will determine the best way to manage your phenylalanine blood levels. It is very important to keep the level of phenylalanine in the blood under control before and during pregnancy.
It is not known if Palynziq is excreted in breast milk or if it affects your baby. Talk to your healthcare provider about the best way to feed your child if you are using Palynziq.
It is not known if Palynziq affects fertility. Animal studies suggest that women may have difficulty getting pregnant if they have abnormally low phenylalanine levels.
Driving and using machines
Palynziq may 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 (23 mg) sodium per pre-filled syringe, ie essentially ‘sodium-free’.
How to use Palynziq
Always use this medicine exactly as your doctor has told you. Consult a doctor if you are unsure.
Palynziq is given as an injection under the skin ( subcutaneous injection ).
Dose
- You start taking the lowest dose of Palynziq. You 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 increase the dose one and/or how often you inject Palynziq slowly. Your doctor will tell you how long to take each dose. By slowly increasing your dose over time, your body can get used to the drug.
- The goal is to reach a daily dose that lowers your phenylalanine level in the blood to within the target range between 120 and 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 level of phenylalanine in the blood.
Examples of steps to reach your goal for phenylalanine levels in the blood
| Palynziq dose and how often to take it | Spray paint |
| 2.5 mg once a week | White piston |
| 2.5 mg twice a week | |
| 10 mg once a week | Green piston |
| 10 mg twice a week | |
| 10 mg four times a week | |
| 10 mg daily | |
| 20 mg daily | Blue piston |
| 40 mg daily(2 injections with 20 mg pre-filled syringe ) 1 | |
| 60 mg daily(3 injections with 20 mg pre-filled syringe ) 1 |
If you need to take more than one injection to get your daily dose, all your injections should be taken at the same time and the injection sites should be at least 5 centimeters apart. Do not divide your daily dose during 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 needs to check your blood phenylalanine level every month to see if the medicine works for you.
Start with Palynziq
- Your healthcare provider will inject Palynziq into you until you (or a healthcare provider) can do it yourself.
- Your doctor will prescribe medicines that you should take before injecting one with Palynziq, such as paracetamol, fexofenadine, and / or ranitidine. These drugs help reduce the symptoms of an allergic reaction.
- A healthcare provider will monitor you for at least 1 hour after you receive Palynziq to see if you get any signs or symptoms of an allergic reaction.
- Your doctor will also prescribe adrenaline injection equipment that you should use if you have a severe allergic reaction. Your healthcare provider will also tell you what signs and symptoms you should be aware of and what to do if you have a severe allergic reaction.
- Your doctor will show you how and when to use the adrenaline injection equipment. Always have it with you.
Continue taking Palynziq
- This medicine comes in pre-filled syringes with 3 different strengths (2.5 mg with white flask, 10 mg with green flask, or 20 mg with blue flask). You may need to use more than one pre-filled syringe to get your prescribed dose. The caregiver tells you which syringe or combination of syringes to use and shows you (or a caregiver) how to inject Palynziq.
- In the operating instructions (section 7 of this leaflet) you will see:
- how to prepare and inject Palynziq
- how to dispose of Palynziq syringes properly after use.
- Your doctor will tell you how long you should continue to take medicines such as paracetamol, fexofenadine, and / or ranitidine before taking Palynziq.
- For at least the first 6 months of treatment with Palynziq, someone must be with you when you inject Palynziq and for at least 1 hour after injection, and observe if you get any signs or symptoms of a severe allergic reaction and, if necessary, give you an injection of adrenaline and contact emergency care.
- Your doctor will train the person in question regarding the signs and symptoms of a severe allergic reaction and how he or she should give an injection of adrenaline.
- Your doctor will tell you if you need someone to observe you 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 have used too much Palynziq, talk to a doctor. More information on what to do based on your symptoms can be found in section 4.
If you forget to use Palynziq
If you forget to take a dose, take the next dose as scheduled. Do not take a double dose of Palynziq to make up for a forgotten dose.
If you stop using Palynziq
If you stop taking Palynziq, the phenylalanine levels in your blood are likely to increase. Talk to your doctor before stopping treatment with Palynziq.
If you have any further questions on the use of this product, ask your doctor.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Allergic reactions are very common ( may affect more than 1 in 10 people) and the severity varies. Symptoms of an allergic reaction may include rash, itching, swelling of the head and face, itchy or watery eyes, coughing, and wheezing. Your doctor may tell you how to treat allergic reactions depending on their severity and prescribe additional medicines to counteract the reaction. Some of these allergic reactions are more severe, as described below, and may require immediate care.
Serious side effects include:
- Sudden severe allergic reactions: (Common – may affect up to 1 in 10 people.) Stop injecting Palynziq if you notice any severe sudden signs of allergy or a combination of the following signs:
- swelling of the face, eyes, lips, mouth, throat, tongue, hands, and / or feet
- difficulty breathing or wheezing
- constriction in the throat or feeling of suffocation
- difficulty swallowing or speaking
- dizziness or fainting
- urinary or fecal incontinence
- fast heartbeat
- hives (itchy rash on the skin) that spreads rapidly
- redness
- severe abdominal pain or stomach cramps, vomiting, or diarrhea.
Use the adrenaline injection equipment according to your doctor’s instructions and seek emergency care. Your doctor will prescribe adrenaline injection equipment for you to use if you have a severe allergic reaction. The doctor trains and instructs you and someone to help you when and how to use adrenaline. Always carry the adrenaline injection kit.
Contact your doctor immediately if you get any of the following:
- A type of allergic reaction called serum sickness, which involves a combination of fever (high body temperature), rash, muscle, and joint pain. (Common – may affect up to 1 in 10 people.)
Other side effects are
Very common: may affect more than 1 in 10 people
- redness, swelling, bruising, tenderness, or pain where you have injected Palynziq
- joint pain
- reduction of complement factor C3 and C4 protein (which is part of your immune system) in blood tests
- allergic reaction
- too low phenylalanine level in blood samples
- headache
- rash
- abdominal pain
- nausea
- vomiting
- hives (raised itchy rash on the skin)
- itching
- thinner hair or hair loss
- cough
- increase in c-reactive protein (CRP) in blood tests (CRP is a protein that shows that you have an inflammation )
- swollen glands in the neck, armpits, or groin
- reddening of the skin
- muscle pain.
Common: may affect up to 1 in 10 people
- joint stiffness
- joint swelling
- muscle stiffness
- skin rash with small nodules
- blisters or flakes on the outer skin layer.
How to store Palynziq
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, and carton after “EXP”. The expiration date is the last day of the specified month.
Store in a refrigerator (2 ° C – 8 ° C). Do not freeze.
If necessary, you can store Palynziq in its sealed tray outside the refrigerator (below 25 ° C) at one time for up to 30 days, protected from heat sources. Make a note of the date the product was removed from the refrigerator on the unopened tray. Once the product has been removed from the refrigerator, it must not be placed in the refrigerator again.
Do not use this medicine if the pre-filled syringe is damaged or if you notice that the solution is discolored, cloudy or if particles are visible.
Use safe syringe disposal procedures. Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.
Contents of the pack and other information
Content declaration
- The active substance is pegvalias.
Each 2.5 mg pre-filled syringe contains 2.5 mg of pegvalias in 0.5 ml of solution.
Each 10 mg pre-filled syringe contains 10 mg of pegvalias in a 0.5 ml solution.
Each 20 mg pre-filled syringe contains 20 mg of pegvalias in a 1 ml solution. - The other ingredients are trometamol, trometamol hydrochloride, sodium chloride (see section 2 for more information), trans-cinnamic acid, water for injections.
What the medicine looks like and the contents of the pack
Palynziq solution for injection ( injection ) is a clear to slightly opalescent, colorless to pale yellow solution. The pre-filled syringe contains an automatic needle guard.
Pre-filled syringe 2.5 mg (white flask):
Each 2.5 mg carton contains 1 pre-filled syringe.
Pre-filled syringe 10 mg (green flask):
Each 10 mg carton contains 1 pre-filled syringe.
20 mg pre-filled syringe (blue flask):
Each 20 mg carton contains 1 or 10 pre-filled syringes.
Not all pack sizes may be marketed.
Marketing Authorization Holder and Manufacturer
BioMarin International Limited
Shanbally, Ringaskiddy
County Cork
Ireland
P43 R298