Pronunciation:

peg-VAL-i-ase - pqpz

Brand Names:

• Palynziq

Dosage Forms:

• Solution

Warnings:

Anaphylaxis has been reported after administration of pegvaliase-pqpz and may occur at any time during treatment. Administer the initial dose of pegvaliase-pqpz 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 ability to recognize signs and symptoms of anaphylaxis and to administer auto-injectable epinephrine, if needed. Prescribe auto-injectable epinephrine. Prior to first dose, instruct the patient and observer (if applicable) on its appropriate use. Instruct the patient to seek immediate medical care upon its use. Instruct patients to carry auto-injectable epinephrine with them at all times during pegvaliase-pqpz treatment. Pegvaliase-pqpz is available only through a restricted program called the Palynziq(TM) REMS .

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Pegvaliase

Uses of This Medicine:

Pegvaliase-pqpz injection is used to lower phenylalanine levels in the blood in adults with phenylketonuria (PKU), who have uncontrolled blood phenylalanine levels with their current treatment.

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

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of pegvaliase-pqpz injection in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies have not been performed on the relationship of age to the effects of pegvaliase-pqpz injection in the geriatric population.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Proper Use of This Medicine:

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.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly and to check for serious unwanted effects.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and requires 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. Keep with you an epinephrine autoinjector pen at all times in case of a severe allergic reaction.

Patients will be given a Palynziq® information card that they should carry with them at all times. This card describes symptoms of an unwanted effect which may occur. Always show this card to any healthcare professional who may treat you.

Side Effects of This Medicine:

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