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PALYNZIQ® (PEGVALIASE-PQPZ)
Injection Access and Support Services

PALYNZIQ is only available through a restricted distribution program called PALYNZIQ REMS. Find out how to enroll your patients with phenylketonuria (PKU) into the program and about dedicated support services from BioMarin to support both you and your patients during treatment.

Enroll Your Patients to Access PALYNZIQ and Benefit from Personalized Support Services

BioMarin is committed to providing personalized support services to help PKU patients start and stay on therapy.

rems

PALZYNZIQ Risk Evaluation and Mitigation Strategy (REMS) Program

Your office must become REMS-certified before you can begin to prescribe PALYNZIQ.

A REMS program is required by the FDA to ensure that the benefits of therapy outweigh the risks. The goal of the PALYNZIQ REMS Program is to mitigate the risk of anaphylaxis.1

Enroll each patient

– Counsel your patient on the risks associated with PALYNZIQ, including the risk of anaphylaxis and the need for auto-injectable epinephrine. Prescribe auto-injectable epinephrine, and instruct patients how to use it and to carry it with them at all times

– Review the following with your patient: REMS Patient Guide, REMS Wallet Card and the REMS Safety Video via the REMS website.

– You and your patient must sign the REMS Patient Enrollment Form

PALYZNIQ REMS Website

Visit PALYNZIQREMS.com or submit signed, completed forms to the PALYNZIQ REMS:

by Fax: 1-866-713-8421

by mail: PALYNZIQ REMS, 200 Pinecrest Plaza, Morgantown, WV 26505

For all other queries please phone: 1-855-758-REMS (1-855-758-7367)

biomarin rare connections

BioMarin RareConnections™

BioMarin RareConnections™ provides patients on PALYNZIQ with personalized Case Management and has Field Reimbursement Managers available to provide reimbursement education to your clinic, product access education, and logistics support.

BioMarin Case Managers provide one-on-one support to address unique product needs.

Personalized financial support

With the help of assigned Case Managers, patients have support throughout the process of securing coverage for their medication, which starts with identifying insurance coverage. If needed, the Case Manager will provide information about financial assistance options that may help patients pay for their treatment.

Specialty pharmacy coordination

Case Managers will work directly with a specialty pharmacy to coordinate delivery of patients’ medications at a date and time that are convenient for them. Case Managers can also provide updates on shipment status.

Ongoing support

BioMarin RareConnections™ Case Managers work closely with clinics and other BioMarin teams to ensure your patients have product support throughout treatment.

Ready to enroll a patient?

Help your patient prescribed PALYNZIQ complete the Patient Authorisation Form to allow BioMarin RareConnections™ to assist them with support and access to treatment.

Enroll Patient Online

Email Form to Patient

Download and Print

BioMarin Co-Pay logo

BioMarin Co-Pay Assistance Program

BioMarin is committed to helping your patients with co-pay or out-of-pocket costs for PALYNZIQ.

With the PALYNZIQ Co-Pay Assistance Program 99% of patients paid $0 out-of-pocket for their prescription.*†‡

 

  • The PALYNZIQ Co-Pay Assistance Program will cover all co-pay costs related to both PALYNZIQ and auto-injectable epinephrine prescriptions, up to the annual maximum, for as long as patients remain on therapy
  • Eligible patients will receive three fills of auto-injectable epinephrine per year at no additional cost

In order to qualify for BioMarin’s Co-Pay Assistance Program, your patients must meet the following criteria:

  • Covered by commercial (private) insurance
  • Not a participant in a federal or state-funded healthcare program, including but not limited to Medicare, Medicaid, VA/DoD, TRICARE, and Medigap
  • Live in the United States or Puerto Rico

*Valid only for those with commercial insurance. Offer not valid for prescriptions eligible to be reimbursed, in whole or in part, by Medicare, Medicaid, or any other federal or state program (including any state prescription drug assistance programs) (eg, VA, DoD, TRICARE), for cash-paying patients, where product is not covered by patient’s commercial insurance, or where plan reimburses you for entire cost of your prescription drug. No claim for reimbursement of the out-of-pocket expense amount covered by the Program shall be submitted to any third-party payer, whether public or private. Offer is not valid where prohibited by law. Valid only in the United States and Puerto Rico. This program is not health insurance. Offer may not be combined with any other rebate, coupon, or offer. Co-payment assistance under the Program is not transferable. BioMarin Pharmaceutical Inc. reserves the right to rescind, revoke, or amend the program without notice. Patient certifies responsibility for complying with applicable limitations, if any, of any commercial insurance and reporting receipt of program rewards, if necessary, to any commercial insurer. This program is subject to termination or modification at any time.
†Based on 2018 and 2019 co-pay program data.

‡The PALYNZIQ Co-Pay Assistance Program will cover up to $16,100 in assistance per calendar year for eligible patients. Some restrictions apply.

PALYNZIQ Financial Support & Co-Pay Assistance

BioMarin RareConnections™ Case Managers can help determine patient eligibility for financial support options.

Learn About Financial Assistance

Email Eligibility to Your Patient

BioMarin Clinical Coordinators

BioMarin Clinical Coordinators are there to support patients’ treatment journeys and help them fit BioMarin treatment into their lifestyles. More specifically, they will provide:

Support throughout therapy

As your patients get started on the treatment plan the clinic sets up, their Clinical Coordinator will be there to answer questions they have about their therapy. Clinical Coordinators are also available for face-to-face or virtual visits.

Education

Patients’ Clinical Coordinators will partner with them to provide information about PKU, their BioMarin therapy, how to take it, and dietary options. They also offer grocery store tours to show patients foods that meet their dietary needs.

Help staying on track

Clinical Coordinators will help provide patients with reminders to stay on track according to the treatment plan the clinic team sets up.

Give Your PKU Patient Access to BioMarin Clinical Coordinators

Complete the BioMarin RareConnections™ Patient Enrollment Form for PALYNZIQ to give your patients access to BioMarin Clinician Coordinators.

This form acts as the prescription for PALYNZIQ, which is only available through specialty pharmacies.

Complete online

Download & Print

Resources for Your PKU Patients

Share these videos with your PKU patients to not only help them understand how to inject PALYNZIQ but also provide key information on side effects, including an important warning for the risk of anaphylaxis.

Please refer to the full Prescribing Information, with Boxed Warning for risk of anaphylaxis, and Medication Guide.

peer-to-peer

Learn from a Peer

As well as providing the programs described above to support your patients throughout their PKU treatment with PALYNZIQ, BioMarin
encourages Peer-to-Peer discussion through its Learn from a Peer
Program.

Request a PALYNZIQ Peer-to-Peer Discussion

Tell us if you would like to request an in-office or virtual
peer-to-peer discussion to learn more about PALYNZIQ by filling in the form below.

Form Here

Terms of Use

By completing and submitting this form, you are indicating that you would like to receive more information related to PALYNZIQ for the treatment of PKU and/or BioMarin’s products and services. BioMarin will not sell, rent, or otherwise distribute your information outside of BioMarin and related third parties. BioMarin will only use the information you enter in accordance with its Privacy Policy.

Play

Guidance for the Use of PALYNZIQ

Hear evidence- and consensus-based recommendations for the use of PALYNZIQ in adults with PKU being discussed. Topics covered include goals of PKU treatment, PALYNZIQ administration and appropriate dosing regimens, and recognizing and responding to adverse events.

17.46

References:

  1. PALYNZIQ [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2020.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: RISK OF ANAPHYLAXIS

  • Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment
  • 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
  • Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use
  • Prescribe auto-injectable epinephrine. 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 PALYNZIQ treatment
  • PALYNZIQ is available only through a restricted program called PALYNZIQ REMS (Risk Evaluation and Mitigation Strategy). Further information, including a list of qualified pharmacies, is available at www.PALYNZIQREMS.com or by telephone at 1-855-758-REMS (1-855-758-7367)

WARNINGS AND PRECAUTIONS

Anaphylaxis

  • Signs and symptoms of anaphylaxis reported include syncope, hypotension, hypoxia, dyspnea, wheezing, chest discomfort/chest tightness, tachycardia, angioedema (swelling of face, lips, eyes, tongue), throat tightness, skin flushing, rash, urticaria, pruritus, and gastrointestinal symptoms (vomiting, nausea, diarrhea)
  • Anaphylaxis generally occurred within 1 hour after injection; however, delayed episodes occurred up to 48 hours after PALYNZIQ administration
  • Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use
  • Anaphylaxis requires immediate treatment with auto-injectable epinephrine. Prescribe auto-injectable epinephrine to all patients receiving PALYNZIQ and instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment. Prior to the first dose, instruct the patient and observer (if applicable) on how to recognize the signs and symptoms of anaphylaxis, how to properly administer auto-injectable epinephrine, and to seek immediate medical care upon its use. Consider the risks associated with auto-injectable epinephrine use when prescribing PALYNZIQ. Refer to the auto-injectable epinephrine prescribing information for complete information
  • Consider the risks and benefits of readministering PALYNZIQ following an episode of anaphylaxis. If the decision is made to readminister PALYNZIQ, administer the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose. Subsequent PALYNZIQ dose titration should be based on patient tolerability and therapeutic response
  • Consider premedication with an H1-receptor antagonist, H2-receptor antagonist, and/or antipyretic prior to PALYNZIQ administration based upon individual patient tolerability

Other Hypersensitivity Reactions

  • Hypersensitivity reactions other than anaphylaxis have been reported in 204 of 285 (72%) patients treated with PALYNZIQ in clinical trials
  • Management of hypersensitivity reactions should be based on the severity of the reaction, recurrence of the reaction, and the clinical judgment of the healthcare provider, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids

ADVERSE REACTIONS

  • The most common adverse reactions (at least 20% of patients in either treatment phase) were injection site reactions, arthralgia, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, nausea, abdominal pain, vomiting, cough, oropharyngeal pain, pruritus, diarrhea, nasal congestion, fatigue, dizziness, and anxiety
  • Of the 285 patients exposed to PALYNZIQ in an induction/titration/maintenance regimen in clinical trials, 44 (15%) patients discontinued treatment due to adverse reactions. The most common adverse reactions leading to treatment discontinuation were hypersensitivity reactions (6% of patients) including anaphylaxis (3% of patients), angioedema (1% of patients), arthralgia (4% of patients), generalized skin reactions lasting at least 14 days (2% of patients), and injection site reactions (1% of patients)
  • The most common adverse reactions leading to dosage reduction were arthralgia (15% of patients), hypersensitivity reactions (9% of patients), injection site reactions (4% of patients), alopecia (3% of patients), and generalized skin reactions lasting at least 14 days (2% of patients)
  • The most common adverse reactions leading to temporary drug interruption were hypersensitivity reactions (14% of patients), arthralgia (13% of patients), anaphylaxis (4% of patients), and injection site reactions (4% of patients)
  • Angioedema and serum sickness: In clinical trials, 22 out of 285 (8%) patients experienced 45 episodes of angioedema (symptoms included: pharyngeal edema, swollen tongue, lip swelling, mouth swelling, eyelid edema, and face edema) occurring independent of anaphylaxis. In clinical trials, serum sickness was reported in 7 out of 285 (2%) patients

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

DRUG INTERACTIONS

Effect of PALYNZIQ on Other PEGylated Products

  • In a single-dose study of PALYNZIQ in adult patients with PKU, two patients receiving concomitant injections of medroxyprogesterone acetate suspension (a formulation containing PEG 3350) experienced a hypersensitivity reaction. One of the two patients experienced anaphylaxis
  • The clinical effects of concomitant treatment with different PEGylated products are unknown. Monitor patients treated with PALYNZIQ and concomitantly with other PEGylated products for hypersensitivity reactions including anaphylaxis

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

  • PALYNZIQ may cause fetal harm when administered to a pregnant woman
  • Advise women who are exposed to PALYNZIQ during pregnancy or who become pregnant within one month following the last dose of PALYNZIQ that there is a pregnancy surveillance program that monitors pregnancy outcomes. Healthcare providers should report PALYNZIQ exposure and encourage these patients to report their pregnancy to BioMarin (1-866-906-6100)
  • Monitor blood Phe levels in breastfeeding women treated with PALYNZIQ

Pediatric Use

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

Geriatric Use

  • Clinical studies of PALYNZIQ did not include patients aged 65 years and older

You are encouraged to report suspected adverse reactions to BioMarin at 1-866-906-6100, or to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information, with Boxed Warning for risk of anaphylaxis, and Medication Guide here.

INDICATION
PALYNZIQ is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe concentrations in adult patients with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.