Pegvaliase

Summary

Pegvaliase (CHEMBL4297802) is an approved enzyme (ATC A16AB19); indicated across 1 condition including phenylketonuria.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB19
• Indications: 1 condition
• Clinical trials: 10

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297802
Name	Pegvaliase
Type	Enzyme
Max phase	4
ATC	A16AB19

Also known as: Bmn 165, BMN 165, BMN-165, Peg pal, Pegvaliasa, Pegvaliase, Pegvaliase pqpz, Pegvaliase-pqpz, Phenylase, Palynziq, PEGVALIASE

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 indication (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
phenylketonuria	4	MONDO:0009861	MONDO:0009861

Clinical trials

Total trials: 10.

Phase distribution

Phase	Trials
Not specified	6
PHASE3	3
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07477691	PHASE4	NOT_YET_RECRUITING	Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
NCT05270837	PHASE3	ACTIVE_NOT_RECRUITING	Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT01819727	PHASE3	COMPLETED	An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT03694353	PHASE3	COMPLETED	Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
NCT04404530	Not specified	RECRUITING	Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
NCT05579548	Not specified	RECRUITING	A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
NCT05813678	Not specified	RECRUITING	A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT06305234	Not specified	RECRUITING	A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT03792451	Not specified	COMPLETED	Nutrition Status of Adults Treated With Pegvaliase
NCT05356377	Not specified	COMPLETED	Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).