PALYNZIQ
Palynziq is a phenylalanine-metabolizing enzyme indicated for adult patients with phenylketonuria (PKU). It is used to reduce blood phenylalanine concentrations in patients who have levels greater than 600 micromol/L despite existing management. This therapy provides a clinical option for adults whose PKU remains uncontrolled on their current treatment regimen.
How PALYNZIQ Works
Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that reduces blood phenylalanine concentrations. It functions by converting phenylalanine into ammonia and trans-cinnamic acid. This mechanism substitutes for the deficient activity of the phenylalanine hydroxylase (PAH) enzyme typically found in patients with phenylketonuria.
Details
- Status
- Prescription
- First Approved
- 2018-05-24
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
PALYNZIQ Approval History
What PALYNZIQ Treats
1 indicationsPALYNZIQ is approved for 1 conditions since its original approval in 2018. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Phenylketonuria
PALYNZIQ Boxed Warning
RISK OF 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 recogn...
WARNING: RISK OF 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 auto‑injectable epinephrine, if needed [see Dosage and Administration ( 2.4 )] . 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 [see Warnings and Precautions ( 5.1 )] . Prescribe auto‑injectable 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 auto‑injectable epinephrine, and to seek immediate medical care upon its use. Instruct patients to carry auto‑injectable epinephrine with them at all times during treatment with Palynziq [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.1 )] . Consider the risks and benefits of readministering Palynziq following an episode of anaphylaxis. If the decision is made to readminister Palynziq, readminister 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 [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.1 )] . Because of the risk of anaphylaxis, P
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Trial Timeline
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Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
PALYNZIQ FDA Label Details
ProIndications & Usage
FDA Label (PDF)Palynziq is indicated to reduce blood phenylalanine concentrations in adult patients 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 Phe concentrations in adult patients with phenylketonuria who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.
WARNING: RISK OF 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 c...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.