Patient Medication Information: uses, side effects, how to take - Palynziq
What is Palynziq used for?
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.
How does Palynziq work?
Palynziq contains the active substance pegvaliase, an enzyme that can break down phenylalanine in the body.
Serious Warnings and Precautions
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What are the ingredients in Palynziq?
Medicinal ingredients: pegvaliase
Non-medicinal ingredients: Sodium chloride, trans-cinnamic acid, trometamol, trometamol hydrochloride, water for injection.
Palynziq comes in the following dosage forms:
Solution for Injection in pre-filled syringe: 2.5 milligrams (mg) (white plunger), 10 mg (green plunger), 20 mg (blue plunger).
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 the list of ingredients for other medicines and in this leaflet (What are the ingredients in Palynziq?)].
To help avoid side effects and ensure proper use, talk to your healthcare professional before you take Palynziq. Talk about any health conditions or problems you may have, including if you:
- Cannot or are not willing to use auto-injectable epinephrine to treat a severe allergic reaction.
- Have ever had a severe allergic reaction to polyethylene glycol (PEG).
Are pregnant, think you may be pregnant, or are planning to become pregnant. Palynziq may harm your unborn baby and use of Palynziq during pregnancy is not recommended. You should use effective birth control when taking Palynziq and for at least 1 month after your last dose of Palynziq. Tell your healthcare professional right away if you become pregnant or think you might be pregnant while taking Palynziq.
- If your phenylalanine levels are too high or too low during pregnancy, this may also affect your unborn baby. You and your healthcare professional can decide the best way for you to manage your blood phenylalanine levels. It is very important to keep your blood phenylalanine levels in the range your healthcare professional recommends during pregnancy.
- There is a pregnancy registry that your healthcare professional can contact if you are pregnant. A pregnancy registry is a large database maintained by BioMarin to collect data on pregnant women taking Palynziq.
- Are breastfeeding or plan to breastfeed. Palynziq may pass into your breast milk. Talk to your healthcare professional about the best way to feed your baby if you take Palynziq.
To obtain additional information, refer to the Patient Medication Materials specific to pregnancy available at www.BioMarin.ca.
Other warnings you should know about:
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.
Give yourself time after taking Palynziq to see how you feel before driving a vehicle or using machinery.
If you stop taking Palynziq treatment, your blood phenylalanine levels are likely to increase. Talk to your doctor before stopping Palynziq treatment.
Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.
The following may interact with Palynziq:
- Injection of other medicines that contain PEG while using Palynziq: Palynziq contains an ingredient called polyethylene glycol (PEG). If you inject Palynziq around the same time as 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.
- There have been no studies testing how Palynziq interacts with other drugs or products.
Patient Education materials have been developed to help you understand allergic reactions and what to do in case of severe allergic reaction that can be found at www.BioMarin.ca.
How to take Palynziq:
Take Palynziq exactly as your doctor or healthcare professional has told you.
Palynziq is given as an injection under the skin (subcutaneous injection).
Be sure that you know from your healthcare professional the dose of Palynziq that you need to use and whether you need to use Palynziq 2.5 mg syringe (white plunger), 10 mg syringe (green plunger), 20 mg syringe (blue plunger), or a combination of syringes to make the dose.
Preparing the dose of Palynziq:
- 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 ≤ 600 micromol/L and does not cause too many side effects. You may need a daily dose of 20 mg, 40 mg, or 60 mg to reach your target blood phenylalanine level.
Example of steps to reach your blood phenylalanine goal
| Palynziq dose and how often to take it | Syringe color |
|---|---|
| 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 centimeters 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 to see if this medicine is working for you and may adjust your dose of Palynziq or ask you to change your diet.
Starting Palynziq:
- Your healthcare professional 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 acetaminophen, fexofenadine, and/or ranitidine. These medicines may help to reduce the symptoms of an allergic reaction.
- A healthcare professional will monitor you for at least 1 hour after you get Palynziq for signs and symptoms of an allergic reaction.
- Your doctor will prescribe an epinephrine injection device to use for any severe allergic reactions. Your healthcare professional will also tell you the 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 epinephrine injection device. Keep it with you at all times.
Continuing Palynziq:
- This medicine comes in prefilled 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 prefilled syringe for your prescribed dose. Your healthcare professional 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” 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 acetaminophen, fexofenadine, and/or ranitidine before you take Palynziq.
For at least the first 6 months of Palynziq treatment after you started receiving the injection at home, you must have an adult trained observer with you when injecting 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 epinephrine and call for emergency medical help.
- Your doctor will train the caregiver/observer on the signs and symptoms of a severe allergic reaction and how to give an injection of epinephrine.
- 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.
Overdose:
| If you think you, or a person you are caring for, have taken too much Palynziq, contact a healthcare professional, hospital emergency department, or regional poison control centre immediately, even if there are no symptoms. |
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Missed Dose:
If you missed a dose of this medication, take it as soon as you remember. If it is almost time for your next dose, skip the missed dose and continue with your next scheduled dose. Go back to your regular dosing schedule. Do not take two doses at the same time.
What are possible side effects from using Palynziq?
These are not all the possible side effects you may have when taking Palynziq. If you experience any side effects not listed here, tell your healthcare professional.
Serious side effects and what to do about them
| Symptom / effect | Talk to your healthcare professional | Stop taking drug and get immediate medical help | |
|---|---|---|---|
| Only if severe | In all cases | ||
| COMMON | |||
Sudden severe allergic reaction:
| ✓ | ✓ | |
| RARE | |||
Serum sickness allergic reaction: combination of
| ✓ | ||
Other side effects with using Palynziq
Very common: may affect more than 1 in 10 people
- Feeling sick (also called nausea)
- Stomach pain
- Vomiting
- Allergic reactions, which can range in severity, and may include the following symptoms: skin rash, itching, swelling of the head or face, itchy or runny eyes, cough, and wheezing
- Skin redness
- Swelling
- Bruising
- Tenderness, or pain where you injected Palynziq
- Joint pain
- Muscle pain
- Headache
- Feeling dizzy
- Cough
- Stuffy nose
- Skin rash
- Hives (raised itchy rash on the skin)
- Itchiness
- Thinning or loss of hair
- Decrease in complement factors C3 and C4 proteins (which are involved in protecting against infections) in blood tests
- Increase in c-reactive protein (CRP) in blood tests (CRP is a protein that indicates that you have inflammation)
- Too low levels of phenylalanine in blood tests
Common (may affect up to 1 in 10 people)
- Swollen glands in the neck, armpit or groin
- Joint swelling
- Joint stiffness
- Muscle stiffness
- Skin rash with small bumps
- Blistering or peeling of the outer layer of the skin
Your healthcare professional will decide when to perform blood tests and will interpret the results.
If you have a troublesome symptom or side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.
Reporting Side Effects
You can report any suspected side effects associated with the use of health products to Health Canada by:
NOTE: Contact your health professional if you need information about how to manage your side effects. The Canada Vigilance Program does not provide medical advice. |
Storage:
- Store in a refrigerator (2 to 8°C). Do not freeze.
- If needed, you may store Palynziq in its sealed tray outside the refrigerator (20 to 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.
- Store in the original carton to protect from light.
- Do not keep Palynziq that is out of date, or that you no longer need.
Keep out of reach and sight of children.
If you want more information about Palynziq:
- Talk to your healthcare professional
- Go to the Patient Information Materials on BioMarin website at www.biomarin.ca
- Find the full product monograph that is prepared for healthcare professionals and includes this Patient Medication Information by visiting the Health Canada website: (https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html; or by calling 1-800-983-4587.