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PKU

What is PKU?

PKU (Phenylketonuria) is an inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). In the U.S., about 17,500 people are living with PKU and approximately 350 babies are born with it each year. This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood.

Clinical manifestations of sustained high Phe levels include a variety of serious neurological and neuropsychological complications, including:

  • Intellectual disability and brain damage
  • Mood disturbances
  • Seizures and tremors
  • Cognitive problems
  • Executive function deficits


“The Pursuit of PKU” details the history of the rare condition, as well as BioMarin’s 15-year commitment to advancing the standard of care for this community, including the introduction of the first and only therapies for PKU.

Diagnosis of PKU

As a result of global newborn screening efforts implemented in the 1960s and early 1970s, virtually all people currently under the age of 40 who live in developed countries were diagnosed during the newborn period.

Managing PKU

For people living with PKU, diet is the first line management, which includes a low-protein diet and specially formulated Phe-free medical foods. When managing the diet it is important to keep the below in mind:

    1. Phenylalanine is a component of all natural protein. Milk and dairy products, meat, fish, chicken, eggs, beans and nuts contain very high Phe levels and must be excluded from the diet. Fruits, vegetables, and breads and pastas also contain Phe, but since the Phe levels in these foods are lower, small amounts may be able to be included in the diet.

    2. Phe-free medical foods provide necessary nutrients (e.g., tyrosine).

There are two FDA-approved treatments for PKU.

  • PALYNZIQ®(pegvaliase-pqpz) Injection, is a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 µmol/L on existing management. (See below for Important Safety Information, including Boxed Warning for a risk of anyaphylaxis).
  • KUVAN® (sapropterin dihydrochloride), a synthetic form of the enzyme cofactor BH4, helps reduce Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU, to be used in conjunction with a Phe-restricted diet.

PALYNZIQ 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.

PALYNZIQ Important Safety Information

BOXED WARNING: RISK OF ANAPHYLAXIS

  • Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment with PALYNZIQ.
  • 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 to administer auto-injectable epinephrine, if needed.
  • Prescribe auto-injectable epinephrine to all patients treated with PALYNZIQ. Prior to the 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 treatment with PALYNZIQ.
  • PALYNZIQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the PALYNZIQ REMS.

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 a restricted program called the PALYNZIQ REMS (Risk Evaluation and Mitigation Strategy). 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 hypersensitivity reactions (severe allergic reactions).

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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What is KUVAN?

Kuvan is a prescription medicine used to lower blood levels of phenylalanine (Phe), in adults and children one month of age and older with a certain type of Phenylketonuria (PKU). Kuvan is used along with a Phe-restricted diet.

What should I tell my doctor before taking Kuvan?

Before you take Kuvan, tell your doctor about all your medical conditions, including if you:

  • are allergic to sapropterin dihydrochloride or any of the ingredients in Kuvan. See the list of ingredients in Kuvan at the end of this leaflet.
  • have poor nutrition or have loss of appetite.
  • are pregnant or plan to become pregnant.
    Pregnancy Exposure Registry: There is a pregnancy exposure registry for women who take Kuvan during pregnancy. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can take part in this registry or contact the registry program at 1-800-983-4587.
  • are breastfeeding or plan to breastfeed. It is not known if Kuvan passes into your breast milk. Talk to your doctor about the best way to feed your baby if you take Kuvan.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, herbal, and dietary supplements. Kuvan and other medicines may interact with each other.
Especially tell your doctor if you take:

  • a medicine that contains levodopa
  • an antifolate medicine
  • sildenafil (Revatio, Viagra), tadalafil (Adcirca, Cialis), vardenafil (Staxyn, Levitra)

Tell your doctor if you are not sure if your medicine is one that is listed above.

Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

How should I take KUVAN?

  • Take Kuvan exactly as your doctor tells you. Your doctor should tell you how much Kuvan to take and when to take it.
  • Your doctor may change your dose of Kuvan depending on how you respond to treatment.
  • Take Kuvan 1 time each day with a meal. It is best to take Kuvan at the same time each day.
  • Kuvan comes as a tablet and powder for oral solution.
    • You can swallow Kuvan tablets whole or dissolve the tablets in water or apple juice. You may also crush the tablets and mix in a small amount of soft food, such as apple sauce or pudding before taking.
    • Be sure that you know what dose of Kuvan powder your doctor prescribed and whether you should use Kuvan 100 mg packets, Kuvan 500 mg packets, or both types of packets to prepare your dose.
    • Open Kuvan powder packets only when you are ready to use them.
    • Kuvan powder for oral solution should be dissolved in water or apple juice. You may also mix the powder for oral solution in a small amount of soft food, such as apple sauce or pudding before taking.
    • See the detailed “Instructions for Use” that comes with Kuvan for information about the correct way to dissolve and take a dose of Kuvan tablets or Kuvan powder for oral solution.
  • It is not possible to know if Kuvan will work for you until you start taking Kuvan. Your doctor will check your blood Phe levels when you start taking Kuvan to see if the medicine is working.
  • During treatment with Kuvan:
    • Any change you make to your diet may affect your blood Phe level. Follow your doctor’s instructions carefully and do not make any changes to your dietary Phe intake without first talking with your doctor. Even if you take Kuvan, if your Phe blood levels are not well controlled, you can develop severe neurologic problems.
    • Your doctor should continue to monitor your blood Phe levels often during your treatment with Kuvan, to make sure that your blood Phe levels are not too high or too low.
    • If you have a fever, or if you are sick, your blood Phe level may go up. Tell your doctor as soon as possible so they can change your dose of Kuvan to help keep your blood Phe levels in the desired range.
  • If you forget to take your dose of Kuvan, take it as soon as you remember that day. Do not take 2 doses in a day.
  • If you take too much Kuvan, call your doctor for advice.

What are the possible side effects of KUVAN?

Kuvan can cause serious side effects, including:

  • Severe allergic reactions. Stop taking Kuvan and get medical help right away if you develop any of these symptoms of a severe allergic reaction:
    • wheezing or trouble breathing
    • coughing
    • feeling lightheaded or you faint/li>
    • flushing
    • nausea
    • rash
  • Inflammation of the lining of the stomach (gastritis) or esophagus (esophagitis). Gastritis or esophagitis can happen with Kuvan and may be severe. Call your doctor right away if you have any of these signs or symptoms:
    • severe upper stomach-area (abdominal) discomfort or pain, nausea and vomiting
    • blood in your vomit or stool
    • black, tarry stools
    • difficulty swallowing
    • loss of appetite
    • pain in the throat
  • Phe levels that are too low. Some children under the age of 7 years who take high doses of Kuvan each day may experience low Phe levels.
  • Too much or constant activity (hyperactivity) can happen with Kuvan. Tell your doctor if you have any signs of hyperactivity, including:
    • fidgeting or moving around too much
    • talking too much

The most common side effects of Kuvan are:

  • headache
  • runny nose and nasal congestion
  • sore throat
  • diarrhea
  • vomiting
  • cough

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of Kuvan. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

How should I store KUVAN?

  • Store Kuvan at room temperature between 68°F to 77°F (20°C to 25°C).
  • Keep Kuvan tablets in the original bottle with the cap closed tightly.
  • Protect from moisture.

Keep Kuvan and all medicines out of the reach of children.

General information about the safe and effective use of KUVAN.
Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use Kuvan for a condition for which it was not prescribed. Do not give Kuvan to other people, even if they have the same symptoms you have. It may harm them. You can ask your pharmacist or doctor for information about Kuvan that is written for health professionals.

What are the ingredients in KUVAN?

Active ingredient: sapropterin dihydrochloride.
Kuvan tablet inactive ingredients: ascorbic acid, crospovidone, dibasic calcium phosphate, D-mannitol, riboflavin, and sodium stearyl fumarate.
Kuvan powder for oral solution inactive ingredients: ascorbic acid, D-mannitol, potassium citrate, and sucralose.

Manufactured by:
BioMarin Pharmaceutical Inc.
Novato, CA 94949

© BioMarin Pharmaceutical Inc. All rights reserved.
Kuvan is a registered trademark of BioMarin Pharmaceutical Inc.

For more information, go to www.kuvan.com or call 1-877-695-8826.

This Patient Information has been approved by the U.S. Food and Drug Administration

Revised:
05/2020

BioMarin

Diseases

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

Products

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

Patient/Physician Support Contact Information

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

Global Medical Information

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