Palynziq
Palynziq (pegvaliase) is injected under the skin to treat phenylketonuria (PKU) in adults who still have high phenylalanine (Phe) even though they've tried other medications for PKU. Once you on the maintenance dose, you only have to take this medication once daily. Because it can put you at high risk for a severe allergic reactions (anaphylaxis) that can be life-threatening, you must register with a REMS program in order to receive this medication. This program will teach you how to recognize a serious reaction and what to do if it happens.
What is Palynziq (pegvaliase)?
What is Palynziq (pegvaliase) used for?
To lower the level of Phe in adults with phenylketonuria (PKU) who have a Phe level greater than 600 micromol/L on other PKU medications
How Palynziq (pegvaliase) works
Palynziq (pegvaliase) is an enzyme replacement therapy. In phenylketonuria (PKU), your body isn't able to make an enzyme (protein) called phenylalanine hydroxylase (PAH). This enzyme is important for breaking down phenylalanine (Phe), which is an essential amino acid.
Palynziq (pegvaliase) replaces the missing PAH for people with PKU. This allows your body to break down Phe.
Drug Facts
What are the side effects of Palynziq (pegvaliase)?
Common Side Effects
- Redness, itching, pain, bruising, rash, swelling, tenderness, and other injection site reactions (74-88%)
- Joint pain (68-74%)
- Headache (36-56%)
- Skin reactions lasting at least 2 weeks (21-41%)
- Nausea (18-31%)
- Stomach pain (14-30%)
- Vomiting (13-30%)
- Cough (9-30%)
- Pain in mouth and throat (13-29%)
- Itching (20-27%)
- Diarrhea (9-27%)
- Nasal congestion (4-27%)
- Feeling tired (13-24%)
- Dizziness (16-21%)
- Anxiety (5-21%)
- Hair loss (5-19%)
Serious Side Effects
- Severe allergic reactions: dizziness, hives, rash, blisters, skin reddening, swelling of the lips or tongue, difficulty breathing, closing of the throat, tightness or discomfort in the chest, vomiting, nausea, diarrhea
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions or angioedema—skin rash, itching or hives, swelling of the face, eyes, lips, tongue, arms, or legs, trouble swallowing or breathing
- Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Palynziq (pegvaliase)
Pros
Given once weekly to once daily
Works well to keep Phe levels low
Available only through a REMS program to make sure it's as safe for you as possible
Can be stored at room temperature for up to 30 days, making it convenient if you're traveling
Cons
Needs to be injected under the skin
Might be difficult for some people to administer correctly
Brand name only, which can be expensive
Hasn't been studied in children under 18 years old or adults 65 years and older
Pharmacist tips for Palynziq (pegvaliase)
Before starting this medication, carefully read the instructions on how to give the injection. Make sure your provider or pharmacist teaches you how to give Palynziq (pegvaliase). Learning to use Palynziq (pegvaliase) correctly will help avoid accidental injuries or medication waste. Contact your provider or pharmacist if you have any questions on how to use this medication.
Palynziq (pegvaliase) can cause sudden severe allergic reactions (anaphylaxis) that can be life-threatening. Make sure to always keep your epinephrine (EpiPen) with you wherever you go. If you noticed your epinephrine (EpiPen) is expired, please replace it as soon as possible
Ask your provider about whether or not you need to maintain a low protein diet while taking Palynziq (pegvaliase). Depending on your Phe levels, you might need to change your diet so your Phe levels don't get too low or too high.
If you have minor allergic reactions after Palynziq (pegvaliase) injections, such as fever or minor rash, let your provider know. They might recommend you to take antihistamines or fever medication, such as acetaminophen (Tylenol), before the injections to help ease symptoms.
Palynziq (pegvaliase) comes as 2.5 mg (white), 10 mg (green), and 20 mg (blue) prefilled syringes. Depending on your dose, you might need to give yourself multiple injections. If you need more than 1 injection per dose, make sure to give the injections at least 2 inches apart from each other.
Store Palynziq (pegvaliase) in the refrigerator and keep the vials in the original carton away from light.
When it's time to give the dose, take the medication out of the refrigerator and allow it to reach room temperature. This will take about 30 minutes. Don't try to warm up the medication any other way (e.g., microwave, under hot water) because you might damage it. You can also store Palynziq (pegvaliase) at room temperature for up to 30 days. Once stored at room temperature, don't put it back in the refrigerator.
Always check to make sure the medication is clear and colorless to pale yellow. If it's cloudy, discolored, or has many particles (solid parts), don't use it. It's okay to use the medication if you see air bubbles, don't try to flick the syringe or push the bubbles out. Contact your or your child's provider or pharmacist to see if it's possible to get a replacement.
Each Palynziq (pegvaliase) syringe provides a single dose of medication. You must throw away any leftover medication after each use, even if there's still some remaining in the syringe. This is because the syringe doesn't contain any preservatives to keep germs and bacteria out of the medication once you open it.
After giving yourself the medication, throw away the syringe and needle in a sharps container or something made of heavy-duty plastic (e.g., empty milk carton, laundry detergent bottle) to lower the risk of needle-stick injuries. Don't throw them away in your trash can. When your sharps container is almost full, close it tightly with the cap. For more information on how to throw away your sharps container safely and guidance for your specific state, visit the FDA's website.
Frequently asked questions about Palynziq (pegvaliase)
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What are the risks and warnings for Palynziq (pegvaliase)?
Palynziq (pegvaliase) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Severe allergic reactions
You might experience severe and sometimes life-threatening allergic reactions after receiving Palynziq (pegvaliase). Most reactions happen within the hour of your injection. You can have an reaction at any time during treatment, even if you've been taking this medication without any problems for years.
When you first start Palynziq (pegvaliase), your provider will watch you give yourself the medication and monitor you for allergic reactions for at least 1 hour after the injection. Make sure you know how to use it and keep it with you at all times. When your provider thinks it's safe, they will train you to give yourself the injections at home. They'll also prescribe you epinephrine (EpiPen). Make sure your caregiver or loved ones also know how to use epinephrine (EpiPen) and can help you use it if you have a severe allergic reaction. If you do experience a serious allergic reaction, you must get your next dose at your provider's office so they can observe you.
If you notice dizziness, a choking sensation, nausea, swelling around your eyes, swelling of the lips or tongue, wheezing, stomach symptoms (e.g., vomiting, nausea, diarrhea), skin flushing, rash, or difficulty breathing after using Palynziq (pegvaliase), give yourself the epinephrine (EpiPen) and call 911 right away.
Palynziq (pegvaliase) REMS program
Because Palynziq (pegvaliase) can cause a life-threatening allergic reaction, it's only available through a restricted program called Risk Evaluation and Mitigation Strategies (REMS) program. Under this program, only providers and pharmacies who are registered and received special training to recognize and treat the allergic reactions can prescribe and dispense this medication. You also need to enroll in this program and be trained on what to do if you have an allergic reaction. Ask your provider if you're not sure how to register for Palynziq (pegvaliase) REMS.
Palynziq (pegvaliase) dosage forms
Typical dosing for Palynziq (pegvaliase)
Your provider will adjust your dose of Palynziq (pegvaliase) depending your Phe level and on if you're having side effects.
The typical starting dose is 2.5 mg of Palynziq (pegvaliase) injected under the skin once weekly for one month. The typical maintenance range is between 20 mg to 40 mg once daily. The maximum dose is 60 mg once daily.
What are alternatives to Palynziq (pegvaliase)?
To lower the level of Phe in adults with phenylketonuria (PKU) who have a Phe level greater than 600 micromol/L on other PKU medications
Used with a Phe-restricted diet to treat high blood phenylalanine (Phe) levels in adults and children one month of age and older
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