Pegvaliase-pqpz (Subcutaneous)

Pegvaliase-pqpz injection is used to reduce phenylalanine levels in the blood of adults with phenylketonuria (PKU) who have high blood phenylalanine levels that are not controlled by their current treatment.

This medicine is only available through a special distribution program called the Palynziq® REMS (Risk Evaluation and Mitigation Strategy) Program.

It is very important that your doctor checks your progress at regular visits to ensure that this medicine is working correctly and to look for serious unwanted effects.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and needs immediate medical attention. Tell your doctor right away if you have a rash, itching, hoarseness, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth after using this medicine. Always keep an epinephrine autoinjector pen with you in case of a severe allergic reaction.

Patients will receive a Palynziq® information card that they should carry at all times. This card describes symptoms of unwanted effects that may occur. Always show this card to any healthcare professional who may treat you.

In deciding to use a medicine, you and your doctor need to consider the risks of the medicine against the benefits it may provide. For this medicine, the following points should be taken into account:

Allergies

Inform your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also, let your healthcare provider know about any other allergies you might have, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric

Studies have not been conducted to understand how pegvaliase-pqpz injection affects children. Its safety and effectiveness in this age group have not been established.

Geriatric

Studies have not been conducted to understand how pegvaliase-pqpz injection affects older adults.

Breast Feeding

There are no adequate studies in women to determine the risk to infants when using this medication while breastfeeding. Consider the potential benefits and risks before using this medication while breastfeeding.

Interactions with Medicines

Some medicines should not be used together at all, while in other cases, two different medicines can be used together even if there may be an interaction. In these situations, your doctor may need to adjust the dose, or take other precautions. Inform your healthcare provider if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Interactions with Food/Tobacco/Alcohol

Certain medicines should not be taken around mealtimes or with specific types of food due to potential interactions. Alcohol or tobacco use with certain medicines may also lead to interactions. Talk to your healthcare provider about how to use your medicine in relation to food, alcohol, or tobacco.

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may happen, if they do, they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

• Cough
• Diarrhea
• Difficulty swallowing
• Dizziness
• Fast heartbeat
• Fever
• Hives, itching, skin rash
• Joint or muscle pain
• Large, hive-like swelling on the face, eyelids, lips, tongue, throat, hands, legs, feet, or genitals
• Nausea
• Numbness or tingling of the face, hands, or feet
• Puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
• Redness and soreness of the eyes
• Sores in the mouth
• Stomach pain
• Swelling of the feet or lower legs
• Tightness in the chest
• Trouble breathing
• Unusual tiredness or weakness
• Vomiting

Rare

• Feeling of discomfort
• Joint swelling
• Swollen lymph glands

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

• Anxiety
• Bleeding, blistering, burning, coldness, discoloration of skin, feeling of pressure, hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site
• Difficulty in moving
• Headache
• Loss or thinning of hair
• Muscle stiffness
• Pain in the mouth and throat
• Stuffy nose

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

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

In the U.S.

• Palynziq

Available Dosage Forms:

• Solution

Therapeutic Class: Endocrine-Metabolic Agent

Pharmacologic Class: Pegvaliase

Find detailed information on biosimilars for this medication.

A nurse or other trained health professional will give you this medicine. It is given through as a shot under your skin, usually in the buttocks, stomach, thighs, or upper arm. You or your caregiver may be trained to prepare and inject this medicine at home. Be sure that you understand how to use the medicine.

Your doctor will give you the first dose of this medicine at the beginning and when restarting the medicine after you had an allergic reaction to the injection. You will need to stay for at least 60 minutes to check for serious unwanted effects.

If you use this medicine at home, you will be shown the body areas where this shot can be given. Use a different body area each time you give yourself a shot. Keep track of where you give each shot to make sure you rotate body areas. Do not inject into skin areas that are tender, red, bruised, hard, burned, inflamed, or has scars, birthmarks, rashes, moles, or tattoos.

If more than one injection is needed, you may inject into the body area at least 2 inches away from each other. The second injection site can be on the same or different part of the body.

It is very important that you understand the requirements of the Palynziq® REMS program, and become familiar with the Palynziq® medication guide and patient instructions. Read and follow these instructions carefully. Ask your doctor if you have any questions. Ask your pharmacist for the medication guide and patient instructions if you do not have one.

Allow the medicine to warm to room temperature for 30 minutes before you use it. Do not warm it in any other way.

Check the liquid in the prefilled syringe. It should be clear or colorless to slightly yellow. Do not use it if it is cloudy, discolored, or has particles in it. Do not shake.

Do not remove the needle cap from the prefilled syringe until you are ready to use it.

You might not use all of the medicine in each prefilled syringe. Use each prefilled syringe only one time. Do not save an open syringe.

You may also take other medicines (eg, allergy medicine, fever medicine, or stomach medicine) before using the injection to prevent serious unwanted effects. You may also have your caregiver or family member stay with you for at least 60 minutes after injecting each dose to check for these effects.

Dosing

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For injection dosage form (solution):For lowering phenylalanine levels in the blood: Adults—The starting dose is usually 2.5 milligrams (mg) injected under the skin once a week for 4 weeks. Your doctor may adjust your dose as needed and tolerated. However, the dose is usually not more than 60 mg injected under the skin once a day for 16 weeks. Children—Use and dose must be determined by your doctor.

Missed Dose

If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

Storage

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store in the refrigerator. Do not freeze.

You may also keep this medicine at room temperature for up to 30 days. Do not return it to the refrigerator once you keep it at room temperature.

Throw away used needles in a hard, closed container that the needles cannot poke through. Keep this container away from children and pets.

• What is Palynziq used to treat?