Palynziq

Palynziq is a prescription medication used to treat phenylketonuria (PKU).

Palynziq is an enzyme which breaks down phenylalanine and serves as a substitute for the enzyme which is lacking in PKU patients.

This medication is available in an injectable form to be given directly under the skin (subcutaneously) by a healthcare professional. Your healthcare provider will decide if you (or your caregiver) are able to give Palynziq injections at home.

Common side effects of Palynziq include injection site reactions (such as redness, itching, pain, bruising, rash, swelling, or tenderness), joint pain, and headache.

Palynziq is a prescription medication used to lower blood levels of Phe (phenylalanine) in adults with PKU (phenylketonuria) who have uncontrolled blood Phe levels above 600 micromol/L (10 mg/dL) on their current treatment. 

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

Serious side effects have been reported with Palynziq. See the “Palynziq Precautions” section.

Common side effects of Palynziq include the following:

• injection site reactions such as redness, itching, pain, bruising, rash, swelling, or tenderness
• joint pain
• headache
• skin reactions that spread on the skin and last at least 14 days, such as itching, rash, or redness
• itching
• nausea
• stomach pain
• mouth and throat pain
• vomiting
• cough
• diarrhea
• feeling very tired
• low levels of phenylalanine in the blood  

This is not a complete list of Palynziq side effects. Ask your doctor or pharmacist for more information.

Tell your doctor if you have any side effects that bother you or that do not go away.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. Especially tell your doctor if you receive

• injections of medroxyprogesterone
• injections of medications that are PEGylated, such as pegadamase (Adagen), pegaspargase (Oncaspar), pegfilgrastim (Neulasta), certolizumab pegol (Cimzia), and naloxegol (Movantik), or pegaptanib (Macugen)  

This is not a complete list of Palynziq drug interactions. Ask your doctor or pharmacist for more information.

Serious side effects have been reported with Palynziq including the following:

Palynziq can cause a severe allergic reaction (anaphylaxis) that may be life-threatening and can happen any time during treatment with Palynziq. Severe allergic reactions are a serious but common side effect of Palynziq.

• You will receive your first injection of Palynziq in a healthcare setting where you will be closely watched for at least 1 hour after your injection for a severe allergic reaction.
• If you have a severe allergic reaction during treatment with Palynziq, you will need to receive an auto-injection of epinephrine immediately and get emergency medical help right away.
• Your healthcare provider will decide if you (or your caregiver) are able to give the Palynziq injections, recognize the signs and symptoms of a severe allergic reaction, give an injection of epinephrine and call for emergency medical help, if needed.
• Your healthcare provider may recommend that an adult observer (or your caregiver) be with you when you give your Palynziq injection, and 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 epinephrine and call for emergency medical help.

Stop injecting Palynziq and get emergency medical care right away if you have any of the following symptoms of a severe allergic reaction during treatment with Palynziq:

• fainting (passing out)
• dizziness or lightheadedness
• sudden confusion
• trouble breathing or wheezing
• chest discomfort or chest tightness
• fast heart rate
• swelling of your face, lips, eyes, or tongue
• throat tightness
• flushed skin
• skin rash, itching, or raised bumps on skin
• nausea, vomiting, or diarrhea
• losing control of urine or stool

Your healthcare provider will prescribe an auto-injectable epinephrine for you and will teach you (or your caregiver) and your observer, if needed, when and how to use it if you have a severe allergic reaction. Keep the auto-injectable epinephrine with you at all times during treatment with Palynziq. Read the Patient Information that comes with the auto-injectable epinephrine that your healthcare provider prescribes for you for more information.

If you have a severe allergic reaction, do not continue to take Palynziq until you talk with your healthcare provider. Tell your healthcare provider that you had a severe allergic reaction. Your healthcare provider will tell you if you can continue treatment with Palynziq.

• Your healthcare provider may prescribe other medicines to take before your Palynziq injection that may help reduce the symptoms of an allergic reaction.
• If your healthcare provider decides that you can continue treatment with Palynziq after a severe allergic reaction, you will receive your next injection of Palynziq in a healthcare setting where you will be closely watched for at least 1 hour after your injection for a severe allergic reaction.

Your healthcare provider will give you a Palynziq Patient Wallet Card that describes symptoms that you (or your caregiver), or your observer, should know that require you to get emergency medical care right away. Carry this card with you at all times during treatment with Palynziq. It is important to show your Palynziq Patient Wallet Card to any other healthcare provider who treats you.

Palynziq is only available through a restricted program called the Palynziq Risk Evaluation and Mitigation Strategy (REMS) Program. Before you can receive Palynziq, you must:

• enroll in this program.
• receive education about the risk of a severe allergic reaction (anaphylaxis) by a healthcare provider certified in the Palynziq REMS to be sure that you understand the risks and benefits of treatment with Palynziq.
• fill the prescription your healthcare provider gives you for the auto-injectable epinephrine and carry it with you at all times during treatment with Palynziq.
• carry the Palynziq Patient Wallet Card with you at all times.

Talk to your healthcare provider for more information about the Palynziq REMS and how to enroll.

Do not take Palynziq if you are allergic to Palynziq or to any of its ingredients. 

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Palynziq, there are no specific foods that you must exclude from your diet when receiving this medication.

Before taking Palynziq, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

• cannot or are not willing to use auto-injectable epinephrine to treat a severe allergic reaction
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed

Tell your healthcare provider about all the medicines you take, including prescription or over-the-counter medicines, vitamins, or herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

In animal studies, pregnant animals were given Palynziq and had some babies born with problems. No well-controlled studies have been done in humans. However, phenylalanine levels that are too high in pregnant women have caused some babies to be born with problems. Therefore, this medication may be used if the potential benefits to the mother outweigh the potential risks to the unborn child.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Palynziq crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using Palynziq.

Receive Palynziq injections exactly as prescribed by your doctor.

This medication is available in an injectable form to be given directly under the skin by a healthcare professional. Your healthcare provider will decide if you (or your caregiver) are able to give the Palynziq injections at home.

Palynziq is usually given once a week for 4 weeks and then slowly increased to a once daily injection.

Your doctor may prescribe you a medication to take before your Palynziq injections to help reduce the symptoms of an allergic reaction. Your doctor will also prescribe you auto-injectable epinephrine and teach you when and how to use it in the case of a severe allergic reaction.

Keep track of how much protein and phenylalanine you eat or drink while taking Palynziq. Follow your doctor's instructions about how much protein and phenylalnine you should have in your diet.

If you miss a dose, skip the missed dose and take your next dose at the regular time. Do not take two doses of Palynziq at the same time.

Receive this medication exactly as prescribed by your doctor.

The dose your doctor recommends may be based on how you respond to this medication.

Your doctor will give you a 2.5 mg injection of Palynziq weekly for 4 weeks. Depending on how you respond to the drug, your doctor will slowly increase your dose over time to a 20 mg injection once daily.

If you take too much Palynziq, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

• Store Palynziq in the refrigerator at 36°F to 46°F (2°C to 8°C).
• If needed, you may store Palynziq at room temperature between 68°F to 77°F (20°C to 25°C) for up to 30 days.
  • Write the date that you remove Palynziq from the refrigerator on the carton.
  • If stored at room temperature, do not put Palynziq back in the refrigerator.
• Keep Palynziq in the original carton to protect from light.
• Do not freeze or shake Palynziq.
• Throw away Palynziq if it has been kept at room temperature for 30 days and has not been used, or after the expiration date on the carton, whichever comes first.
• Keep Palynziq and all medicines out of the reach of children.

WARNING: RISK OF ANAPHYLAXIS

• Anaphylaxis has been reported after administration of Palynziq and may occur at any time during treatment.
• Administer the initial dose of Palynziq under the supervision of a healthcare provider equipped to manage anaphylaxis, and closely observe patients for at least 60 minutes following injection. Prior to self‑injection, confirm patient competency with self‑administration, and patient’s and observer’s (if applicable) ability to recognize signs and symptoms of anaphylaxis and administer auto‑injectable epinephrine, if needed.
• Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during Palynziq treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after Palynziq administration, should be able to administer auto‑injectable epinephrine, and call for emergency medical support upon its use.
• Prescribe auto‑injectable epinephrine to all patients treated with Palynziq. Prior to the first dose, instruct the patient and observer (if applicable) how to recognize the signs and symptoms of anaphylaxis, how to properly administer auto‑injectable epinephrine, and to seek immediate medical care upon its use. Instruct patients to carry auto‑injectable epinephrine with them at all times during treatment with Palynziq.
• Consider the risks and benefits of readministering Palynziq following an episode of anaphylaxis. If the decision is made to readminister Palynziq, readminister the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose.
• Because of the risk of anaphylaxis, Palynziq is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS.