Pegvaliase-pqpz

What is Pegvaliase-pqpz?

Pegvaliase-pqpz (Palynziq) is a phenylalanine (Phe)‑metabolizing enzyme lower blood levels of phenylalanine in adults with PKU (phenylketonuria).

What are the uses of this medicine?

• This medicine is used to lower blood levels of phenylalanine in adults with PKU (phenylketonuria) who have uncontrolled blood phenylalanine levels above 600 micromol/L (10 mg/dL) on their current treatment.

How does this medicine work?

• Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans‑cinnamic acid.
• It substitutes for the deficient phenylalanine hydroxylase (PAH) enzyme activity in patients with PKU and reduces blood phenylalanine concentrations.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• Monitor patients treated with Palynziq and concomitantly with other PEGylated products for hypersensitivity reactions including anaphylaxis.

Is this medicine FDA approved?

• It was approved for use in the United States in 2018.

How should this medicine be used?

• Obtain baseline blood phenylalanine concentration before initiating treatment.

Recommended Dosage

• The recommended initial dosage is 2.5 mg subcutaneously once weekly for 4 weeks.
• Titrate the dosage in a step-wise manner over at least 5 weeks based on tolerability to achieve a dosage of 20 mg once daily. See full prescribing information for titration regimen.
• Assess patient tolerability, blood Phe concentration, and dietary protein and Phe intake throughout treatment.
• Consider increasing the dosage to 40 mg once daily in patients who have been on 20 mg once daily continuously for at least 24 weeks and who have not achieved blood Phe control (blood phenylalanine concentration less than or equal to 600 micromol/L).
• Consider increasing the dosage to a maximum of 60 mg once daily in patients who have been on 40 mg once daily continuously for at least 16 weeks and who have not achieved blood Phe control.
• Discontinue Palynziq in patients who have not achieved an adequate response after 16 weeks of continuous treatment with the maximum dosage of 60 mg once daily.
• Reduce the dosage and/or modify dietary protein and Phe intake, as needed, to maintain blood Phe concentrations within a clinically acceptable range and above 30 micromol/L.

Blood Phenylalanine Monitoring and Diet

• Obtain blood Phe concentrations every 4 weeks until a maintenance dosage is established.
• Periodically monitor blood Phe concentrations during maintenance therapy.
• Counsel patients to monitor dietary protein and Phe intake, and adjust as directed by their healthcare provider.

Premedication

• For hypersensitivity reactions, consider premedication with an H1‑receptor antagonist, H2‑receptor antagonist, and/or antipyretic prior to Palynziq administration based upon individual patient tolerability

Administration

• Your healthcare provider will give you the Palynziq injection until they decide that you (or your caregiver) can give it. See “What is the most important information I should know about Palynziq?”
• Your healthcare provider may prescribe medicine for you to take before your Palynziq injection to help reduce the symptoms of an allergic reaction.
• If your healthcare provider decides that you (or your caregiver) can give your Palynziq injections, you (or your caregiver) will be shown how to prepare and give your Palynziq injections. See the “Instructions for Use” for detailed instructions on how to prepare and give an injection of Palynziq.
• Use Palynziq exactly as your healthcare provider tells you to. Your healthcare provider will tell you how much Palynziq to inject and when to inject it.
• Palynziq comes in prefilled syringes with 3 different strengths (2.5 mg, 10 mg, or 20 mg). You may need more than 1 Palynziq prefilled syringe for your prescribed dose.
• Monitor the amount of protein and phenylalanine that you eat or drink. Your healthcare provider may change the amount of protein and phenylalanine you should have in your diet during treatment with Palynziq, depending on the levels of phenylalanine in your blood. Follow your healthcare provider’s instructions about the amount of protein and phenylalanine you should have in your diet.
• If you miss a dose, take your next dose at the regular time. Do not take two doses of Palynziq to make up for a missed dose.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Injection: 2.5 mg/0.5 mL, 10 mg/0.5 mL, and 20 mg/mL in a single-dose prefilled syringe

This medicine is available in fallowing brand namesː

• Palynziq

What side effects can this medication cause?

The most common side effects of this medicine include:

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

Palynziq may cause serious side effects, including: Palynziq can cause a severe allergic reaction (anaphylaxis) that may be life-threatening and can happen any time during treatment with Palynziq which may include:

• 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 stools

What special precautions should I follow?

• Anaphylaxis has been reported after administration of Palynziq and may occur at any time during treatment.
• Management should be based on the severity of the reaction, recurrence, and clinical judgement, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids.
• Prescribe auto‑injectable epinephrine to all patients treated with Palynziq.
• Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during Palynziq treatment.
• 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.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• Palynziq may cause fetal harm when administered to a pregnant woman.
• Limited available data with pegvaliase-pqpz use in pregnant women are insufficient to inform a drug-associated risk of adverse developmental outcomes.

Can this medicine be used in children?

• The safety and effectiveness of Palynziq in pediatric patients have not been established.

What are the active and inactive ingredients in this medicine?

• Active ingredients: pegvaliase-pqpz
• Inactive ingredients: sodium chloride, trans-cinnamic acid, tromethamine, and tromethamine hydrochloride

Who manufactures and distributes this medicine?

• Manufactured by: BioMarin Pharmaceutical Inc. Novato, CA 94949 US License No. 1649

What should I know about storage and disposal of this medication?

• 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.

• Dailymed label info on Pegvaliase-pqpz
• FDA Pegvaliase-pqpz

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