PALYNZIQ (pegvaliase-pqpz)
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.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2018-05-24
- Patent Cliff
- 2025
- Revenue
- $115M (Q4-2025)
- 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
PALYNZIQ Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to PALYNZIQ
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07477691 | 165-401 2021-005058-27 | Ph 4 | not yet recruiting | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) |
| NCT05270837 PEGASUS | 165-306 | Ph 3 | active not recruiting | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria |
| NCT03694353 results posted | 165-304 | Ph 3 | completed | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
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
Indications & Usage
FDA Label (PDF)PALYNZIQ is indicated for the treatment of Phenylketonuria.
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...
Pro Intelligence Preview
Deep insights for PALYNZIQ
Revenue Insights
- • Q4-2025: $115M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2025
- • Generic/biosimilar risk
Trial Analysis
- • 3 total trials
- • Stage: Expansion
Competitive Landscape
- • 4 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment