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For assistance, please call
1-833-PKU-CARE (1-833-758-2273)
Monday-Friday, 8AM-8PM ET
For assistance, please call 1-866-906-6100

Frequently asked questions

Rare diseases can bring uncommon challenges, and everyone’s treatment journey is unique
With BioMarin RareConnections™, you don’t have to face it alone. Your BioMarin team will guide you along your treatment journey, working one-to-one with you to address your unique needs to gain access to treatment. Below are some common questions and answers that might be helpful for you.

About BioMarin RareConnections

BioMarin RareConnections is a program that provides a wide array of support services for BioMarin products to help you understand insurance coverage and gain access to treatment. This may include providing information about financial assistance options and working with your healthcare team to coordinate delivery of BioMarin products for your treatment.
Our dedicated and experienced Case Managers will provide guidance on how you can gain access to BioMarin therapies by:
  • Helping you understand your insurance coverage and financial assistance options
  • Providing you with ongoing product support for BioMarin therapies
  • Coordinating with a specialty pharmacy to deliver your medication at a date and time that is convenient for you
Once you enroll in BioMarin RareConnections, you will be assigned a dedicated experienced Case Manager who understands your unique needs related to treatment. Your Case Manager can help you determine your insurance coverage and, if necessary, identify financial assistance options for your BioMarin treatment.
To enroll in BioMarin RareConnections and get connected with your Case Manager, start by completing the Patient Consent Form.
In order for BioMarin to provide product support services, you will need to provide consent to both your healthcare provider and BioMarin:
  • Your healthcare provider needs your written consent to release your protected health information (PHI) to BioMarin
  • BioMarin also needs your written consent to share your information (including genetic testing information) with other providers to assist you with accessing services that support your treatment
Once you give consent, your healthcare provider will provide BioMarin with the information from your medical records needed to enroll you in BioMarin RareConnections. BioMarin RareConnections will help you work with your insurance provider to seek reimbursement for your care and will help you with other case management services.
A patient or an authorized representative for the patient must sign the authorization form.
If you choose not to sign the authorization form, BioMarin will not be able to provide BioMarin RareConnections support in connection with any BioMarin treatment.
BioMarin will use your protected health information (PHI) to enroll you in BioMarin RareConnections, which provides a number of case management services that help support your treatment plan (for example, insurance navigation support). BioMarin RareConnections will work with you, your healthcare provider, and your health insurance company to help with coverage related to your treatment. If you do not already have insurance coverage, BioMarin RareConnections can help you find insurance options.
The Patient Consent Form is valid from the date of signature for 10 years, or a shorter time if required by applicable state law, or until you cancel the authorization.
You can cancel the authorization at any time by submitting a written notification to BioMarin RareConnections.
Connect with a Case Manager by email or call 1-866-906-6100. Once you have been enrolled in BioMarin RareConnections, your dedicated Case Manager will also reach out to you with an introduction and will provide you with their direct contact info. Your Case Manager will be an ongoing resource, providing product support services to help you start and continue on treatment.

PALYNZIQ® (pegvaliase-pqpz) Injection Indication and Important Safety Information
IMPORTANT WARNING: RISK OF ANAPHYLAXIS

KUVAN® (sapropterin dihydrochloride) Indication and Important Safety Information

What is PALYNZIQ?

PALYNZIQ® (Pal-lin-zeek) (pegvaliase-pqpz) is a prescription medication used to lower blood levels of phenylalanine (Phe) 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 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 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

Pregnancy Surveillance Program
There is a pregnancy surveillance program for females who take PALYNZIQ during pregnancy, or who become pregnant while receiving PALYNZIQ or within 1 month after their last dose of PALYNZIQ. The purpose of this program is to collect information about the health of you and your baby while taking PALYNZIQ. Talk to your healthcare provider about how you can take part in this program or call BioMarin at 1-866-906-6100

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.

Please see full Prescribing Information, including an important warning for risk of anaphylaxis, and the Medication Guide

What is KUVAN?

KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in people with a certain type of Phenylketonuria (PKU). KUVAN is to be used along with a Phe-restricted diet.

What is the most important information I should know about 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 signs or symptoms of a severe allergic reaction:
    • Wheezing or trouble breathing
    • Coughing
    • Feeling lightheaded or you faint
    • Feeling flushed
    • Nausea
    • Rash
  • Inflammation of the lining of the stomach (gastritis). 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
  • Phe levels that are too low—some children under the age of 7 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

What should I tell my doctor before I take 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
  • Have poor nutrition or have a loss of appetite
  • Are pregnant or plan to become pregnant
  • 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, and 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? 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
  • Take KUVAN exactly as your doctor tells you
  • You can swallow KUVAN tablets whole or dissolve the tablets in water or apple juice
  • KUVAN powder for oral solution should be dissolved in water or apple juice
  • 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 blood Phe 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 he or she 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

The most common side effects of KUVAN are: headache, runny nose and nasal congestion, sore throat, diarrhea, vomiting, and cough.
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 the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100.

Please read the full Prescribing Information by clicking here.

PALYNZIQ® (pegvaliase-pqpz) Injection Indication and Important Safety Information
IMPORTANT WARNING: RISK OF ANAPHYLAXIS

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 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

Pregnancy Surveillance Program
There is a pregnancy surveillance program for females who take PALYNZIQ during pregnancy, or who become pregnant while receiving PALYNZIQ or within 1 month after their last dose of PALYNZIQ. The purpose of this program is to collect information about the health of you and your baby while taking PALYNZIQ. Talk to your healthcare provider about how you can take part in this program or call BioMarin at 1-866-906-6100

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.

Please see full Prescribing Information, including an important warning for risk of anaphylaxis, and the Medication Guide

KUVAN® (sapropterin dihydrochloride) Indication and Important Safety Information

What is KUVAN?

KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in people with a certain type of Phenylketonuria (PKU). KUVAN is to be used along with a Phe-restricted diet.

What is the most important information I should know about 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 signs or symptoms of a severe allergic reaction:
    • Wheezing or trouble breathing
    • Coughing
    • Feeling lightheaded or you faint
    • Feeling flushed
    • Nausea
    • Rash
  • Inflammation of the lining of the stomach (gastritis). 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
  • Phe levels that are too low—some children under the age of 7 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

What should I tell my doctor before I take 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
  • Have poor nutrition or have a loss of appetite
  • Are pregnant or plan to become pregnant
  • 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, and 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? 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
  • Take KUVAN exactly as your doctor tells you
  • You can swallow KUVAN tablets whole or dissolve the tablets in water or apple juice
  • KUVAN powder for oral solution should be dissolved in water or apple juice
  • 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 blood Phe 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 he or she 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

The most common side effects of KUVAN are: headache, runny nose and nasal congestion, sore throat, diarrhea, vomiting, and cough.

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 the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100.

Please read the full Prescribing Information by clicking here.