This website is intended for healthcare professionals practicing in the US.

This website is intended for healthcare professionals practicing in the US.

RESET THE STANDARD FOR PHE CONTROL

PALYNZIQ® (pegvaliase-pqpz) Injection is the first FDA-approved therapy that can lower Phe levels across PKU types, regardless of baseline diet.1,2*

PALYNZIQ offers substantial Phe reduction, regardless of baseline diet1-3

The Phase 3 PRISM trials enrolled 261 patients. 118 of these patients were randomized to and received at least one dose of 20 mg.1†

Most patients achieved at least one Phe level ≤360 µmol/L1

Time to achieve this Phe level in the majority of adults (89/118)1,3

Many patients achieved at least one Phe level ≤120 µmol/L1

Time to achieve this Phe level in the majority of adults (79/118)3

Interested in learning more?

Connect with a BioMarin representative for a detailed presentation.

PALYNZIQ IS BACKED BY A WELL-ESTABLISHED SAFETY PROFILE

Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment1

PALYNZIQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the PALYNZIQ REMS.

Hypersensitivity reactions other than anaphylaxis were reported in 204/285 (72%) patients. Management should be based on the severity of the reaction, recurrence, and clinical judgment, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids.1

15% of patients (44 of 285) discontinued treatment due to an adverse event.1

The most common adverse reactions leading to discontinuation were hypersensitivity reactions (6%), including anaphylaxis (3%), angioedema (1%), arthralgia (4%), generalized skin reactions lasting at least 14 days (2%), and injection site reactions (1%).1

Effective management of the most common adverse reactions is possible with1:

  • Premedications, including H1 and H2 antagonists and antipyretics
  • Medications, including NSAIDs, glucocorticoids, antihistamines, corticosteroids, and autoinjectable epinephrine, as needed

Rates of the 3 most common adverse reactions decreased from induction/titration to maintenance†† despite increasing dose1,5

Adverse Reactions Chart Adverse Reactions Chart

Of all patients, only 1 in 10 experienced anaphylaxis, with all episodes resolving without sequelae1

*Monitor patients’ dietary protein and phenylalanine intake throughout treatment with PALYNZIQ and counsel them on how to adjust their dietary intake, as needed, based on blood phenylalanine concentrations.
†Of the 261 patients who enrolled in Study 301, 54 (21%) patients discontinued treatment during Study 301, 4 patients completed Study 301 and did not continue to Study 165-302 (referred to as Study 302, NCT01889862], 152 patients continued to the eligibility period of Study 302, and 51 patients continued directly from Study 301 into the long-term treatment period of Study 302.1
‡Of the 118 patients from Study 301 with a pre-treatment baseline blood phenylalanine concentration greater than 600 micromol/L who were randomized to and received at least one dose of 20 mg once daily, 21% (25/118) of patients discontinued the study prior to achieving ≤360 µmol/L. Of those, 10 patients discontinued due to adverse events. 3% (4/118) did not achieve ≤360 mmol/L before study completion.1,3
§The majority of those patients (86/89) achieved ≤360 µmol/L by 36 months.3
Of the 118 patients from Study 301 with a pre-treatment baseline blood phenylalanine concentration greater than 600 micromol/L who were randomized to and received at least one dose of 20 mg once daily, 24% (28/118) of patients discontinued the study prior to achieving ≤120 µmol/L. 11 patients did not achieve ≤120 µmol/L before study completion.1,3
#The 25th percentile of patients who achieved ≤120 µmol/L did so by 7 months; the 75th percentile, by 18 months.3
**Injection site reactions can include redness, itching, pain, bruising, rash, swelling, or tenderness.1
††Maintenance phase defined as when subjects reached stable dose for 8 weeks.1
‡‡Hypersensitivity, including anaphylaxis.1
§§Maintenance, all doses includes patients on placebo and PALYNZIQ <20 mg.1,5
¶¶27 of 29 patients who had anaphylaxis were tested for anti-pegvaliase-pqpz lgE antibodies, which recognize the PEGylated protein product. Of those 27 patients, 26 tested negative. The one patient who screened positive for anti-pegvaliase-pqpz lgE had insufficient sample to confirm lgE positivity. This patient tested negative for anti-pegvaliase-pqpz lgE at routine visits before and after the anaphylaxis episode.1

References: 1. PALYNZIQ [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2020. 2. Burton B, Sacharow S, Northrup H, et al. Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies. Poster presented at: SSIEM Annual Symposium; August 30-September 2, 2022; Freiburg, Germany. 3. Data on file. BioMarin Pharmaceutical Inc. 4. Thomas J, Levy H, Amato S, et al, for the PRISM investigators. Pegvaliase for the treatment of phenylketonuria: results of a long-term phase 3 clinical trial program (PRISM). Mol Genet Metab. 2018;124(1):27-38. doi:10.1016/j.ymgme.2018.03.006 5. Sacharow S, Northrup H, Whitehall KB, et al. Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies. Presented at the International Congress of Inborn Errors of Metabolism (ICIEM); November 21-24, 2021; Sydney, Australia.

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.