Avalglucosidase Alfa

Summary

Avalglucosidase Alfa (CHEMBL4594320) is an approved enzyme (ATC A16AB22); indicated across 4 conditions including disorder of glycogen metabolism.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB22
• Indications: 4 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594320
Name	Avalglucosidase Alfa
Type	Enzyme
Max phase	4
ATC	A16AB22

Also known as: Avalglucosidasa alfa, Avalglucosidase alfa, Avalglucosidase alfa ngpt, Avalglucosidase alfa-ngpt, GZ-402666, Gz402666, GZ402666, Nexviadyme, Nexviazyme, neoGAA, AVALGLUCOSIDASE 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

4 indications (2 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
disorder of glycogen metabolism	2	MONDO:0002412	MONDO:0002412

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE4	2
PHASE3	2
PHASE2	2
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05164055	PHASE4	ACTIVE_NOT_RECRUITING	Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
NCT06666413	PHASE4	RECRUITING	China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
NCT04910776	PHASE3	ACTIVE_NOT_RECRUITING	Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
NCT02782741	PHASE3	COMPLETED	Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
NCT03019406	PHASE2	ACTIVE_NOT_RECRUITING	A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
NCT02032524	PHASE2	COMPLETED	Avalglucosidase Alfa Extension Study
NCT01898364	PHASE1	COMPLETED	Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
NCT05734521	Not specified	RECRUITING	Avalglucosidase Alfa Pregnancy Study

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).