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
Side effects of Palynziq (pegvaliase)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
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%)
Palynziq (pegvaliase) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- 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
The following Palynziq (pegvaliase) 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.
Common questions about Palynziq (pegvaliase)
Normally, your body makes an enzyme (protein) called phenylalanine hydroxylase (PAH). PAH is needed to break down phenylalanine (Phe), which is an essential amino acid that you get from food. People with phenylketonuria (PKU) can't make PAH. This causes Phe levels to get too high, which can cause brain problems. PKU is typically screened and diagnosed in babies because it's important to catch it early. Signs and symptoms of PKU include vomiting, weakness, and a skin rash. It's even possible for babies with PKU to have a delay in development. Palynziq (pegvaliase) treats PKU by acting as a replacement for PAH and helping the body break down Phe.
To see if Palynziq (pegvaliase) is working, your provider will monitor your Phe levels and your diet to make sure Palynziq (pegvaliase) is working as best as possible for you. Your provider will slowly raise your dose over the course of about 2 months to a maintenance dose. You might need to take this medication for 6 months or more for your provider to see if it's working well for you.
Since Palynziq (pegvaliase) can put you at risk for severe allergic reaction (anaphylaxis), it's only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS program. All providers who prescribe Palynziq (pegvaliase), pharmacies that dispense Palynziq (pegvaliase), and people who take Palynziq (pegvaliase) must be registered with the REMS program and educated about this serious allergic reaction. Under this program, your provider must also prescribe an epinephrine (EpiPen) and train you on how to use it.
You can give the injection under the skin in the stomach (at least 2 inches away from your belly button), front middle of the thighs, back of arms, or buttocks. Never inject the medication into a muscle or vein. To lower the risk of bruising and irritation, rotate between different injection sites each time so that the same area isn't used repeatedly. Don't inject into moles, scars, birthmarks, bruises, rashes, or areas where skin is hard, tender, red, damaged, burned, inflamed, or tattooed to avoid damaging your skin.
Let your provider know right away if you're pregnant. Palynziq (pegvaliase) isn't recommended in pregnancy because it can potentially cause harm to your unborn baby. At the same time, high Phe level can also harm your unborn baby. If you're pregnant or become pregnant within 1 month after the last dose of Palynziq (pegvaliase), contact your provider right away so they can check you and your baby's health.
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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
Typical dosage 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.
