Pegunigalsidase Alfa

Summary

Pegunigalsidase Alfa (CHEMBL4297801) is an approved enzyme (ATC A16AB20); indicated across 1 condition including fabry disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB20
• Indications: 1 condition
• Clinical trials: 9

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297801
Name	Pegunigalsidase Alfa
Type	Enzyme
Max phase	4
ATC	A16AB20

Also known as: Elfabrio, Pegunigalsidasa alfa, Pegunigalsidase alfa, Pegunigalsidase alfa-iwxj, Prx-102, PRX-102, PEGUNIGALSIDASE ALFA

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
Fabry disease	4	MONDO:0010526	MONDO:0010526

Clinical trials

Total trials: 9.

Phase distribution

Phase	Trials
PHASE3	4
PHASE2/PHASE3	2
Not specified	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05710692	PHASE2/PHASE3	RECRUITING	Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
NCT06328608	PHASE2/PHASE3	RECRUITING	A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
NCT02921620	PHASE3	WITHDRAWN	Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
NCT03180840	PHASE3	COMPLETED	Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
NCT03566017	PHASE3	COMPLETED	Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
NCT03614234	PHASE3	COMPLETED	Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
NCT01678898	PHASE1/PHASE2	COMPLETED	Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
NCT07109375	Not specified	NOT_YET_RECRUITING	Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
NCT04552691	Not specified	APPROVED_FOR_MARKETING	Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

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).