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Drug overview for PALYNZIQ (pegvaliase-pqpz):
Generic name: pegvaliase-pqpz (peg-VAL-i-ase)
Drug class: Phenylketonuria (PKU) Treatment Agents
Therapeutic class: Metabolic Modifiers
Pegvaliase-pqpz is a phenylalanine-metabolizing enzyme composed of biosynthetic (recombinant DNA origin) phenylalanine ammonia lyase (PAL) conjugated with polyethylene glycol (PEG).
No enhanced Uses information available for this drug.
Generic name: pegvaliase-pqpz (peg-VAL-i-ase)
Drug class: Phenylketonuria (PKU) Treatment Agents
Therapeutic class: Metabolic Modifiers
Pegvaliase-pqpz is a phenylalanine-metabolizing enzyme composed of biosynthetic (recombinant DNA origin) phenylalanine ammonia lyase (PAL) conjugated with polyethylene glycol (PEG).
No enhanced Uses information available for this drug.
DRUG IMAGES
PALYNZIQ 2.5 MG/0.5 ML SYRINGE
The following indications for PALYNZIQ (pegvaliase-pqpz) have been approved by the FDA:
Indications:
Phenylketonuria
Professional Synonyms:
Classical phenylketonuria
Indications:
Phenylketonuria
Professional Synonyms:
Classical phenylketonuria
The following dosing information is available for PALYNZIQ (pegvaliase-pqpz):
Dosage of pegvaliase should be individualized based on patient tolerability, blood phenylalanine concentrations, and dietary protein and phenylalanine intake. (See Dosage and Administration: General.)
The dosing regimen of pegvaliase for the treatment of phenylketonuria (PKU) consists of an induction, titration, and maintenance phase. The recommended initial adult dosage of pegvaliase-pqpz is 2.5 mg once weekly by subcutaneous injection for 4 weeks.
The dosage should be increased slowly, as tolerated, over at least 5 weeks up to a target maintenance dosage of 20 mg once daily. The manufacturer provides a suggested schedule for dosage titration (see Table 1); however, dosage should be titrated based on individual response and tolerability. Patients who experience adverse effects may require slower titration to achieve their target maintenance dosage.
Table 1: Recommended Pegvaliase-pqpz Dosage Titration Regimen
Treatment Phase Dosage Duration Induction 2.5 mg once weekly 4 weeks Titration 2.5 mg twice weekly 1 week 10 mg once weekly 1 week 10 mg twice weekly 1 week 10 mg four times per 1 week week 10 mg once daily 1 week Maintenance 20 mg once daily 24 weeks
A therapeutic response may not be achieved until an effective maintenance dosage is attained; the lowest effective and tolerated maintenance dosage should be used. If a response is not obtained (i.e., blood phenylalanine concentrations do not decrease by at least 20% from baseline or remain elevated at a concentration greater than 600 mcmol/L) after 24 weeks of treatment with pegvaliase-pqpz 20 mg once daily, dosage may be increased to 40 mg once daily. If blood phenylalanine concentrations still do not decrease by at least 20% from baseline or remain elevated at a concentration greater than 600 mcmol/L after 16 weeks of treatment at the maximum dosage of 40 mg once daily, therapy should be discontinued.
If blood phenylalanine concentrations decrease to less than 30 mcmol/L during titration or maintenance therapy with pegvaliase, dosage may be reduced and/or dietary intake of protein and/or phenylalanine modified to maintain blood phenylalanine concentrations within a clinically acceptable range above 30 mcmol/L.
If anaphylaxis occurs and a decision is made to continue pegvaliase therapy, subsequent dosage should be titrated based on patient tolerability and therapeutic response. (See Anaphylaxis under Warnings/Precautions: Sensitivity Reactions, in Cautions.)
The dosing regimen of pegvaliase for the treatment of phenylketonuria (PKU) consists of an induction, titration, and maintenance phase. The recommended initial adult dosage of pegvaliase-pqpz is 2.5 mg once weekly by subcutaneous injection for 4 weeks.
The dosage should be increased slowly, as tolerated, over at least 5 weeks up to a target maintenance dosage of 20 mg once daily. The manufacturer provides a suggested schedule for dosage titration (see Table 1); however, dosage should be titrated based on individual response and tolerability. Patients who experience adverse effects may require slower titration to achieve their target maintenance dosage.
Table 1: Recommended Pegvaliase-pqpz Dosage Titration Regimen
Treatment Phase Dosage Duration Induction 2.5 mg once weekly 4 weeks Titration 2.5 mg twice weekly 1 week 10 mg once weekly 1 week 10 mg twice weekly 1 week 10 mg four times per 1 week week 10 mg once daily 1 week Maintenance 20 mg once daily 24 weeks
A therapeutic response may not be achieved until an effective maintenance dosage is attained; the lowest effective and tolerated maintenance dosage should be used. If a response is not obtained (i.e., blood phenylalanine concentrations do not decrease by at least 20% from baseline or remain elevated at a concentration greater than 600 mcmol/L) after 24 weeks of treatment with pegvaliase-pqpz 20 mg once daily, dosage may be increased to 40 mg once daily. If blood phenylalanine concentrations still do not decrease by at least 20% from baseline or remain elevated at a concentration greater than 600 mcmol/L after 16 weeks of treatment at the maximum dosage of 40 mg once daily, therapy should be discontinued.
If blood phenylalanine concentrations decrease to less than 30 mcmol/L during titration or maintenance therapy with pegvaliase, dosage may be reduced and/or dietary intake of protein and/or phenylalanine modified to maintain blood phenylalanine concentrations within a clinically acceptable range above 30 mcmol/L.
If anaphylaxis occurs and a decision is made to continue pegvaliase therapy, subsequent dosage should be titrated based on patient tolerability and therapeutic response. (See Anaphylaxis under Warnings/Precautions: Sensitivity Reactions, in Cautions.)
Pegvaliase is administered by subcutaneous injection. Pegvaliase is intended for use under the guidance and supervision of a clinician; however, the drug may be self-administered if the clinician determines that the patient and/or their caregiver is competent to safely administer the drug after appropriate training and with medical follow-up as necessary. Pegvaliase-pqpz injection is commercially available in single-use prefilled syringes equipped with a 26-gauge, 1/2-inch needle.
The injection is a clear to slightly opalescent, colorless to pale yellow solution. Prior to administration, pegvaliase solution should be inspected visually for particulate matter or discoloration; if either is present, the solution should be discarded. Prefilled syringes of pegvaliase-pqpz injection contain no preservatives and are intended for single use only.
Pegvaliase-pqpz prefilled syringes should be stored at 2-8degreesC in the original carton to protect the drug from light. The injection should not be frozen or shaken, and should not be used if subjected to these conditions. If necessary, the prefilled syringes may be stored at room temperature in the original carton for up to 30 days; the syringes should not be returned to the refrigerator once they have been stored at room temperature.
Subcutaneous injections of pegvaliase should be administered into the middle front of the thigh or abdomen (except within 2 inches of the navel); if a caregiver is administering the injection, the top of the buttocks or back of the upper arm also may be used. Injection sites should be rotated. If multiple injections are needed for a single dose, the injection sites should be separated by at least 2 inches.
Injections should not be made into areas where the skin is tender, swollen, bruised, inflamed, red, burned, damaged, tattooed, or hard, or into moles, scars, or birthmarks. If a dose of pegvaliase is missed, the next dose should be administered at the regularly scheduled time; 2 doses should not be administered to make up for a missed dose.
The injection is a clear to slightly opalescent, colorless to pale yellow solution. Prior to administration, pegvaliase solution should be inspected visually for particulate matter or discoloration; if either is present, the solution should be discarded. Prefilled syringes of pegvaliase-pqpz injection contain no preservatives and are intended for single use only.
Pegvaliase-pqpz prefilled syringes should be stored at 2-8degreesC in the original carton to protect the drug from light. The injection should not be frozen or shaken, and should not be used if subjected to these conditions. If necessary, the prefilled syringes may be stored at room temperature in the original carton for up to 30 days; the syringes should not be returned to the refrigerator once they have been stored at room temperature.
Subcutaneous injections of pegvaliase should be administered into the middle front of the thigh or abdomen (except within 2 inches of the navel); if a caregiver is administering the injection, the top of the buttocks or back of the upper arm also may be used. Injection sites should be rotated. If multiple injections are needed for a single dose, the injection sites should be separated by at least 2 inches.
Injections should not be made into areas where the skin is tender, swollen, bruised, inflamed, red, burned, damaged, tattooed, or hard, or into moles, scars, or birthmarks. If a dose of pegvaliase is missed, the next dose should be administered at the regularly scheduled time; 2 doses should not be administered to make up for a missed dose.
| DRUG LABEL | DOSING TYPE | DOSING INSTRUCTIONS |
|---|---|---|
| PALYNZIQ 2.5 MG/0.5 ML SYRINGE | Maintenance | Adults inject 0.5 milliliter (2.5 mg) by subcutaneous route once weekly in the abdomen, thigh, upper buttocks, or outer upper arm (rotate site) for 4 weeks |
| PALYNZIQ 20 MG/ML SYRINGE | Maintenance | Adults inject 1 milliliter (20 mg) by subcutaneous route once daily in the abdomen, thigh, upper buttocks, or outer upper arm (rotate site) |
| PALYNZIQ 10 MG/0.5 ML SYRINGE | Maintenance | Adults inject 0.5 milliliter (10 mg) by subcutaneous route once daily in the abdomen, thigh, upper buttocks, or outer upper arm (rotate site) |
No generic dosing information available.
The following drug interaction information is available for PALYNZIQ (pegvaliase-pqpz):
There are 0 contraindications.
There are 0 severe interactions.
There are 0 moderate interactions.
The following contraindication information is available for PALYNZIQ (pegvaliase-pqpz):
Drug contraindication overview.
The manufacturer states there are no known contraindications to the use of pegvaliase-pqpz.
The manufacturer states there are no known contraindications to the use of pegvaliase-pqpz.
There are 0 contraindications.
There are 1 severe contraindications.
Adequate patient monitoring is recommended for safer drug use.
| Severe List |
|---|
| Pregnancy |
There are 0 moderate contraindications.
The following adverse reaction information is available for PALYNZIQ (pegvaliase-pqpz):
Adverse reaction overview.
Adverse effects reported in at least 15% of patients receiving pegvaliase in clinical studies include injection site reactions, arthralgia (e.g., back pain, musculoskeletal pain, pain in extremities, neck pain), hypersensitivity reactions, pruritus, headache, generalized skin reactions lasting at least 14 days, nausea, vomiting, abdominal pain, diarrhea, oropharyngeal pain, cough, nasal congestion, fatigue, anxiety, dizziness, and alopecia.
Adverse effects reported in at least 15% of patients receiving pegvaliase in clinical studies include injection site reactions, arthralgia (e.g., back pain, musculoskeletal pain, pain in extremities, neck pain), hypersensitivity reactions, pruritus, headache, generalized skin reactions lasting at least 14 days, nausea, vomiting, abdominal pain, diarrhea, oropharyngeal pain, cough, nasal congestion, fatigue, anxiety, dizziness, and alopecia.
There are 4 severe adverse reactions.
| More Frequent | Less Frequent |
|---|---|
|
Hypersensitivity drug reaction |
Anaphylaxis Angioedema Serum sickness |
| Rare/Very Rare |
|---|
| None. |
There are 17 less severe adverse reactions.
| More Frequent | Less Frequent |
|---|---|
|
Acute abdominal pain Arthralgia Cough Diarrhea Fatigue Headache disorder Injection site sequelae Nausea Pain in oropharynx Pruritus of skin Skin rash Vomiting |
Alopecia Nasal congestion Skin inflammation Symptoms of anxiety Urticaria |
| Rare/Very Rare |
|---|
| None. |
The following precautions are available for PALYNZIQ (pegvaliase-pqpz):
Safety and efficacy of pegvaliase have not been established in pediatric patients.
Contraindicated
Severe Precaution
Management or Monitoring Precaution
Contraindicated
| None |
Severe Precaution
| None |
Management or Monitoring Precaution
| None |
Blood phenylalanine concentrations should be monitored during pregnancy, and dosage of pegvaliase or dietary intake of protein or phenylalanine adjusted to maintain blood phenylalanine concentrations greater than 30 mcmol/L. (See Pregnancy under Warnings/Precautions: Specific Populations, in Cautions.) Pegvaliase may cause fetal harm based on animal findings. There are no adequate data in pregnant women.
In pregnant rabbits receiving pegvaliase at exposures 7.5 times the human exposure at the maximum recommended dosage, embryofetal toxicity (e.g., decreased fetal weight, increased resorptions), teratogenicity (e.g., skeletal and soft tissue abnormalities), and maternal toxicity were observed. In rats receiving pegvaliase at exposures 4.2-19.4
times the human exposure at the maximum recommended dosage, skeletal variations (but no malformations) and maternal toxicity were observed; adverse postnatal effects (e.g., decreased pup weight and litter size, delayed sexual maturation) also occurred. Poorly controlled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of fetal loss (miscarriage), major birth defects (e.g., microcephaly, cardiac malformations), reduced fetal growth, and adverse effects on postnatal development (e.g., intellectual disability, low IQ). In addition, blood phenylalanine concentrations less than 30 mcmol/L during pregnancy can increase the risk of adverse fetal outcomes.
Blood phenylalanine concentrations should be closely monitored during pregnancy and dosage of pegvaliase or dietary phenylalanine and protein intake adjusted as necessary. Blood phenylalanine concentrations should be maintained between 120-360 mcmol/L during pregnancy and for 3 months before conception to decrease the risk of adverse fetal effects. The manufacturer has established a pregnancy registry to collect data on pregnant women treated with pegvaliase and women who become pregnant within 1 month following their last dose of the drug. For additional information or to enroll in the registry, patients may call 1-866-906-6100.
In pregnant rabbits receiving pegvaliase at exposures 7.5 times the human exposure at the maximum recommended dosage, embryofetal toxicity (e.g., decreased fetal weight, increased resorptions), teratogenicity (e.g., skeletal and soft tissue abnormalities), and maternal toxicity were observed. In rats receiving pegvaliase at exposures 4.2-19.4
times the human exposure at the maximum recommended dosage, skeletal variations (but no malformations) and maternal toxicity were observed; adverse postnatal effects (e.g., decreased pup weight and litter size, delayed sexual maturation) also occurred. Poorly controlled blood phenylalanine concentrations before and during pregnancy are associated with an increased risk of fetal loss (miscarriage), major birth defects (e.g., microcephaly, cardiac malformations), reduced fetal growth, and adverse effects on postnatal development (e.g., intellectual disability, low IQ). In addition, blood phenylalanine concentrations less than 30 mcmol/L during pregnancy can increase the risk of adverse fetal outcomes.
Blood phenylalanine concentrations should be closely monitored during pregnancy and dosage of pegvaliase or dietary phenylalanine and protein intake adjusted as necessary. Blood phenylalanine concentrations should be maintained between 120-360 mcmol/L during pregnancy and for 3 months before conception to decrease the risk of adverse fetal effects. The manufacturer has established a pregnancy registry to collect data on pregnant women treated with pegvaliase and women who become pregnant within 1 month following their last dose of the drug. For additional information or to enroll in the registry, patients may call 1-866-906-6100.
Pegvaliase is distributed into milk in rats. Increased pup mortality and impaired weight gain have been observed when the drug was administered to lactating rats. It is not known whether pegvaliase is distributed into human milk or whether the drug affects milk production or the nursing infant.
The benefits of breast-feeding should be considered along with the importance of pegvaliase to the woman and any potential adverse effects on the breast-fed infant from the drug or underlying maternal condition. Pegvaliase may decrease phenylalanine concentrations in human milk. Blood phenylalanine concentrations should be monitored in breast-feeding women receiving pegvaliase therapy.
The benefits of breast-feeding should be considered along with the importance of pegvaliase to the woman and any potential adverse effects on the breast-fed infant from the drug or underlying maternal condition. Pegvaliase may decrease phenylalanine concentrations in human milk. Blood phenylalanine concentrations should be monitored in breast-feeding women receiving pegvaliase therapy.
Pegvaliase has not been studied in geriatric patients 65 years of age or older.
The following prioritized warning is available for PALYNZIQ (pegvaliase-pqpz):
WARNING: This medication has caused serious allergic reactions which can be life-threatening. This reaction can happen at any time during treatment. Get medical help right away if you notice any symptoms of a serious allergic reaction, including: flushing/rash, itching/swelling (especially of the face, tongue, throat), severe dizziness, trouble breathing, fast heartbeat, fainting, chest discomfort/tightness, nausea, vomiting, or diarrhea.
To be sure that you do not have a serious reaction, you will need to stay in a health care setting (such as a doctor's office) for at least 1 hour after taking this drug for the first time so your doctor can carefully monitor you and provide medical help if needed. After the first injection, you and your doctor may decide you can use this medication at home. Your health care provider will instruct you and/or your caregiver how to inject this medication the right way.
Your doctor will also prescribe another medication (epinephrine) in case you have a serious allergic reaction outside the health care setting. Read the Patient Information Leaflet for this medication provided by your pharmacist. Learn ahead of time when and how to use your brand of epinephrine.
If you have to use it, get medical help as soon as you have used it. To receive pegvaliase in the United States, you must understand, agree to, and carefully follow the requirements of the REMS Program for this medication. If you live in Canada or any other country, consult your doctor and pharmacist for your country's regulations.
WARNING: This medication has caused serious allergic reactions which can be life-threatening. This reaction can happen at any time during treatment. Get medical help right away if you notice any symptoms of a serious allergic reaction, including: flushing/rash, itching/swelling (especially of the face, tongue, throat), severe dizziness, trouble breathing, fast heartbeat, fainting, chest discomfort/tightness, nausea, vomiting, or diarrhea.
To be sure that you do not have a serious reaction, you will need to stay in a health care setting (such as a doctor's office) for at least 1 hour after taking this drug for the first time so your doctor can carefully monitor you and provide medical help if needed. After the first injection, you and your doctor may decide you can use this medication at home. Your health care provider will instruct you and/or your caregiver how to inject this medication the right way.
Your doctor will also prescribe another medication (epinephrine) in case you have a serious allergic reaction outside the health care setting. Read the Patient Information Leaflet for this medication provided by your pharmacist. Learn ahead of time when and how to use your brand of epinephrine.
If you have to use it, get medical help as soon as you have used it. To receive pegvaliase in the United States, you must understand, agree to, and carefully follow the requirements of the REMS Program for this medication. If you live in Canada or any other country, consult your doctor and pharmacist for your country's regulations.
The following icd codes are available for PALYNZIQ (pegvaliase-pqpz)'s list of indications:
| Phenylketonuria | |
| E70.0 | Classical phenylketonuria |
| E70.1 | Other hyperphenylalaninemias |
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