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PALYNZIQ (pegvaliase-pqpz) Injection for PKU

Important Safety Information:
WARNING: RISK OF ANAPHYLAXIS

PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life-threatening and can happen any time during treatment with PALYNZIQ.
For more information, read the Important Safety Information below or see the full Prescribing Information at PALYNZIQ.com.

PALYNZIQ (pegvaliase-pqpz) Injection is the first FDA-approved enzyme substitution therapy designed to address the underlying cause of phenylketonuria (PKU), a rare and genetic brain-threatening condition marked by an inability to break down phenylalanine (Phe). PALYNZIQ™ is a once-daily self-administered therapy, clinically proven to reduce blood Phe levels in adults with PKU who have uncontrolled blood Phe levels on existing management.

Left untreated, high levels of Phe can become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, impacting the way a person thinks, feels, and acts. PKU requires daily lifelong management and affects just 350 US newborns each year.
PALYNZIQ substitutes a PEGylated version of the enzyme phenylalanine ammonia lyase for the deficient PAH enzyme.

PALYNZIQ At-a-Glance

  • PALYNZIQ™ is the first enzyme therapy approved for adults living with PKU.
  • Potential to alter the course of PKU management by limiting the need for a Phe-restricted diet
  • Manufactured and commercialized by BioMarin

PALYNZIQ REMS: PALYNZIQ is available only through the PALYNZIQ REMS, a restricted distribution program.

Regulatory Status

In May 2018, PALYNZIQ was approved by the U.S. Food and Drug Administration. Additionally, on March 28, 2018, the European Medicines Agency accepted BioMarin’s submission of a Marketing Authorization Application.

Commercialization Plan

PALYNZIQ is manufactured and commercialized by BioMarin in the United States.

Additional Resources

Palynziq.com
palynziqREMS.com
PKU.com
NPKUA.org

Please see Important Safety Information and Boxed Warning below

PALYNZIQ REMS: PALYNZIQ is available only through the PALYNZIQ REMS, a restricted distribution program.

Indication

PALYNZIQ (Pal-lin-zeek) (pegvaliase-pqpz) Injection 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. You should discuss the potential benefits and risks of PALYNZIQ with your healthcare provider.

Important Safety Information

What is the most important information I should know about PALYNZIQ?
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
  • Your healthcare provider will prescribe 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
  • If you have a severe allergic reaction during treatment with PALYNZIQ, you will need to receive an 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 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:

  • 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

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. Your healthcare provider will tell you if you can continue treatment with PALYNZIQ.

People taking PALYNZIQ have also experienced allergic reactions other than anaphylaxis. Talk to your healthcare provider if you experience any allergic reactions when taking PALYNZIQ.

PALYNZIQ REMS: PALYNZIQ is available only through the PALYNZIQ REMS, a restricted distribution program. Talk to your healthcare provider about the PALYNZIQ REMS and how to enroll.

What should I tell my healthcare provider BEFORE starting PALYNZIQ?

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

Before injecting PALYNZIQ, talk to your healthcare provider right away if you cannot or will not use auto-injectable epinephrine to treat a severe allergic reaction. If you are pregnant or plan to become pregnant while taking PALYNZIQ, talk to your healthcare provider to discuss the risks and benefits of taking PALYNZIQ during pregnancy to you and your unborn baby. If you are breastfeeding or plan to breastfeed, talk to your healthcare provider about the best way to feed your baby if you take PALYNZIQ.
Before injecting PALYNZIQ, read the Medication Guide and Instructions for Use that come with your PALYNZIQ injection.

What should I watch for AFTER starting PALYNZIQ?

PALYNZIQ may cause serious side effects, including:

  • Severe allergic reactions (anaphylaxis)
  • Other allergic reactions to PALYNZIQ can happen during treatment with PALYNZIQ. Contact your healthcare provider right away if you have any of the following symptoms of an allergic reaction including: rash, itching, or swelling of the face, lips, eyes, or tongue. Your healthcare provider may change your dose of PALYNZIQ, stop your treatment with PALYNZIQ for a period of time, or prescribe medicine for you to take before your PALYNZIQ injection to help reduce the symptoms of an allergic reaction

The most common side effects of PALYNZIQ include injection site reactions (such as redness, itching, pain, bruising, rash, swelling, or tenderness), joint pain, headache, skin reactions that spread 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, and low levels of Phe in your blood.
These are not all of the possible side effects of PALYNZIQ. Speak with your healthcare provider right away about any side effects.

Important notes
Blood Phe testing and diet

  • Your healthcare provider will monitor your blood Phe concentrations during PALYNZIQ treatment
  • Monitor the amount of protein and Phe that you eat or drink. Your healthcare provider may change the amount of protein and Phe you should have in your diet during treatment with PALYNZIQ, depending on the levels of Phe in your blood. Follow your healthcare provider’s instructions about the amount of protein and Phe you should have in your diet

Missed dose

  • If a dose is missed, take your next dose as scheduled and do not take two doses of PALYNZIQ to make up for the missed dose

You may report side effects to BioMarin at 1-877-695-8826 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

PALYNZIQ
Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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