Palynziq (pegvaliase-pqpz) is a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria.These highlights do not include all the information needed to use PALYNZIQ safely and effectively. See full prescribing information for PALYNZIQ.
PALYNZIQ (pegvaliase-pqpz) injection, for subcutaneous use
Initial U.S. Approval: 2018
WARNING: RISK OF ANAPHYLAXIS See full prescribing information for complete boxed warning.
Anaphylaxis has been reported after administration of Palynziq and may occur at any time during treatment. (5.1)
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. (2.4)
Prescribe auto?injectable epinephrine. Prior to 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. (2.4, 5.1)
Palynziq is available only through a restricted program called the Palynziq REMS. (5.2)
INDICATIONS AND USAGE
Palynziq is a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. (1)
DOSAGE AND ADMINISTRATION
Obtain baseline blood phenylalanine concentration before initiating treatment.
The recommended initial dosage is 2.5 mg subcutaneously once weekly for 4 weeks.
Titrate the dosage in a step-wise manner over at least 5 weeks based on tolerability to achieve a dosage of 20 mg subcutaneously once daily. See full prescribing information for titration regimen.
Assess patient tolerability, blood phenylalanine concentration, and dietary protein and phenylalanine intake throughout treatment.
Consider increasing the dosage to a maximum of 40 mg subcutaneously once daily in patients who have been on 20 mg once daily continuously for at least 24 weeks and who have not achieved either a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration less than or equal to 600 micromol/L.
Discontinue Palynziq in patients who have not achieved at least a 20% reduction in blood phenylalanine concentration from pre-treatment baseline or a blood phenylalanine concentration less than or equal to 600 micromol/L after 16 weeks of continuous treatment with the maximum dosage of 40 mg once daily.
Reduce the dosage and/or modify dietary protein and phenylalanine intake, as needed, to maintain blood phenylalanine concentrations within a clinically acceptable range and above 30 micromol/L.
Blood Phenylalanine Monitoring and Diet (2.2)
Obtain blood phenylalanine concentrations every 4 weeks until a maintenance dosage is established.
After a maintenance dosage is established, periodically monitor blood phenylalanine concentrations.
Counsel patients to monitor dietary protein and phenylalanine intake, and adjust as directed by their healthcare provider.
Premedication (2.3, 5.1, 5.3)
Consider premedication for hypersensitivity reactions.
Administration Instructions (2.4)
Rotate injection sites. If more than one injection is needed for a single dose, the injection sites should be at least 2 inches away from each other.
DOSAGE FORMS AND STRENGTHS
Injection: 2.5 mg/0.5 mL, 10 mg/0.5 mL, and 20 mg/mL in a single-dose prefilled syringe. (3)
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions, Other than Anaphylaxis: Management should be based on the severity of the reaction, recurrence, and clinical judgement, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids. (5.3)
Most common adverse reactions (at least 20% in either treatment phase) are: injection site reactions, arthralgia, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, pruritus, nausea, abdominal pain, oropharyngeal pain, vomiting, cough, diarrhea, and fatigue. (6.1)
To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Effect of Palynziq on Other PEGylated Products: Monitor for hypersensitivity reactions, including anaphylaxis, with concomitant treatment. (7.1)
USE IN SPECIFIC POPULATIONS
Pregnancy: May cause fetal harm. (8.1)
See 17 for PATIENT COUNSELING INFORMATION and Medication Guide.