pharmacopeia
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Boxed warning

ANAPHYLAXIS Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment [see Warnings and Precautions (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 administer epinephrine, if needed [see Dosage and Administration (2.5) ] . 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 epinephrine, and call for emergency medical support upon its use [see Warnings and Precautions (5.1) ] . Prescribe epinephrine to all patients treated with PALYNZIQ. Prior to the first dose, instruct the patient and observer (if applicable) how to recognize the signs and symptoms of anaphylaxis, how to properly administer epinephrine, and to seek immediate medical care upon its use.

Mechanism of action

Sourced from openFDA

Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid. It substitutes for the deficient phenylalanine hydroxylase (PAH) enzyme activity in patients with PKU and reduces blood phenylalanine concentrations.

Indications

Sourced from openFDA
  • PALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. PALYNZIQ is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.

Contraindications

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  • None. None.contraindicated

Dosage & administration

Sourced from openFDA

Obtain baseline blood Phe concentration before initiating treatment. ( 2.1 ) Consider premedication for hypersensitivity reactions. ( 2.1 , 5.1 , 5.3 ) The recommended initial dosage for PALYNZIQ is 2.5 mg subcutaneously once weekly for 4 weeks. ( 2.2 ) See the Full Prescribing Information for titration, maintenance, discontinuation, and dose reduction. ( 2.2 ) See Full Prescribing Information for dosage and administration modifications due to anaphylaxis. ( 2.3 ) Obtain blood Phe concentrations every 4 weeks until a maintenance dosage is established. ( 2.4 ) Periodically monitor blood Phe concentrations during maintenance therapy and monitor dietary protein and phenylalanine intake throughout treatment. ( 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. ( 2.5 ) 2.1 Important Recommendation Prior to PALYNZIQ Treatment Treatment with PALYNZIQ should be managed by a healthcare provider experienced in the management of PKU. Obtain baseline blood phenylalanine concentration before initiating treatment. Consider premedication with an H 1 -receptor antagonist, H 2 -receptor antagonist, and/or antipyretic based upon individual patient tolerability [see Warnings and Precautions (5.1 , 5.3) ] . 2.2 Recommended Dosage and Administration Induction The recommended initial induction dosage for PALYNZIQ is 2.5 mg subcutaneously once weekly for 4 weeks. Administer the initial dose under the supervision of a healthcare provider [see Dosage and Administration (2.5) ].

Warnings & precautions

Sourced from openFDA

Hypersensitivity Reactions, Other than Anaphylaxis : Management should be based on the severity of the reaction, recurrence, and clinical judgement. ( 5.3 ) Injection Site Infections : Serious injection site infections, including abscess, cellulitis, necrosis, and ulcer have been reported. Instruct patients to contact their healthcare provider if signs or symptoms of an infection develop, persist, or worsen. ( 5.4 ) Hypophenylalaninemia : Some PKU patients treated with PALYNZIQ have experienced hypophenylalaninemia; monitor blood Phe levels periodically during treatment. ( 5.5 ) 5.1 Anaphylaxis In PALYNZIQ Studies 1, 2, and 3 with an induction/titration/maintenance dosage regimen, 10% (29/285) of PALYNZIQ-treated patients experienced a total of 42 anaphylaxis episodes [see Adverse Reactions (6.1) and Clinical Pharmacology (12.6) ]. The exposure-adjusted rate of anaphylaxis was highest during the induction and titration phases (0.25 episodes/person-years; 5% of PALYNZIQ-treated patients with at least one episode) and decreased in the maintenance phase (0.05 episodes/person-years; 7% of PALYNZIQ-treated patients with at least one episode). Signs and symptoms of anaphylaxis reported in PALYNZIQ-treated patients included 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).

Adverse reactions

Sourced from openFDA

The following serious adverse reactions are discussed below and in other sections of labeling: Anaphylaxis [see Warnings and Precautions (5.1) ] Other Hypersensitivity Reactions [see Warnings and Precautions (5.3) ] Injection Site Infections [see Warnings and Precautions (5.4) ] Hypophenylalaninemia [see Warnings and Precautions (5.5) ] 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, nausea, abdominal pain, vomiting, cough, oropharyngeal pain, pruritus, diarrhea, nasal congestion, fatigue, dizziness, and anxiety. ( 6.1 ) Most common adverse reactions in patients who are 12 to less than 18 years of age (at least 20% and greater than in control) are: injection site reactions, arthralgia, headache, pyrexia, hypersensitivity reactions, dizziness, nausea, vomiting, fatigue, and pain in extremity. ( 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. 6.1 Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Use in specific populations

Sourced from openFDA

8.1 Pregnancy Risk Summary Available data from pharmacovigilance and published literature with pegvaliase-pqpz use in pregnant women have not identified a clear association with major birth defects, miscarriage or other adverse maternal or fetal outcomes (see Data ) . There are risks to the fetus associated with poorly controlled phenylalanine concentrations in women with PKU during pregnancy including increased risk for miscarriage, major birth defects (including microcephaly and major cardiac malformations), intrauterine fetal growth retardation, and future intellectual disability with low IQ; therefore, phenylalanine concentrations should be closely monitored in women with PKU during pregnancy (see Clinical Considerations and Data ). Advise pregnant women of the potential risks to the fetus. A reproduction study in pregnant rabbits without PKU treated with pegvaliase-pqpz demonstrated a high incidence of fetal malformations throughout the skeletal system, and in kidneys, lungs, and eyes. Embryo-fetal toxicity (increased resorptions and reduced fetal weight) was also observed. These effects occurred at 5 times the maximum recommended daily dose and were associated with strong signs of maternal toxicity, including marked reductions in weight gain and food consumption, and death.

Pharmacokinetics

Sourced from openFDA
Metabolism
The pharmacokinetics of pegvaliase-pqpz exhibit high inter-patient and intra-patient variability due to the heterogeneity of the immune response in adult patients with PKU. Higher antibody titers correlated with higher apparent clearance of pegvaliase-pqpz.

Approval history

Sourced from openFDA
  • May 24, 2018BLABLA761079Biomarin Pharm

FAERS reports

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Reference statistics only. FAERS reports are voluntarily submitted and are not incidence rates, safety signals, or causal evidence. Counts reflect reporting volume — how often a reaction was reported, not how often it occurs. For decision-grade use, consult openFDA and the FAERS Public Dashboard directly.
7,922 total reports matchedLatest report Share = reports listing the reaction ÷ total matched reports. Rows can sum to >100% because a single report often lists multiple reactions.
  1. 1Arthralgia1,35617%
  2. 2Injection Site Reaction88511%
  3. 3Injection Site Erythema7219.1%
  4. 4Injection Site Swelling5316.7%
  5. 5Rash4886.2%
  6. 6Urticaria4826.1%
  7. 7Headache4265.4%
  8. 8Injection Site Pain4145.2%
  9. 9Fatigue3684.6%
  10. 10Injection Site Pruritus3534.5%
  11. 11Hypersensitivity3334.2%
  12. 12Pruritus3304.2%
  13. 13Off Label Use3224.1%
  14. 14Dizziness3194.0%
  15. 15Anaphylactic Reaction3184.0%

Clinical trials

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The 10 most recently updated of 20 ClinicalTrials.gov registrations naming Pegvaliase as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

Frequently asked questions

How does Pegvaliase work?
Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid. It substitutes for the deficient phenylalanine hydroxylase (PAH) enzyme activity in patients with PKU and reduces blood phenylalanine concentrations.
What is Pegvaliase used for?
According to FDA labeling, Pegvaliase carries indications including: PALYNZIQ is indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. PALYNZIQ is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood phenylalanine concentrations in adult and pediatric patients 12 years of age and older with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
What class of drug is Pegvaliase?
Pegvaliase is classified as Enzymes, Phenylalanine Metabolizing Enzyme, Enzyme Interactions.
What are the brand names for Pegvaliase?
Pegvaliase is marketed under brand names including Palynziq.
What are the contraindications for Pegvaliase?
Pegvaliase labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.
Note. Data for pegvaliase is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.

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