Cipaglucosidase Alfa

Summary

Cipaglucosidase Alfa (CHEMBL4594329) is an approved enzyme (ATC A16AB23); indicated across 3 conditions including disorder of glycogen metabolism.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB23
• Indications: 3 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594329
Name	Cipaglucosidase Alfa
Type	Enzyme
Max phase	4
ATC	A16AB23

Also known as: AT GAA, ATB-200, ATB200, Cipaglucosidase alfa, Cipaglucosidase alfa-atga, Pombiliti, CIPAGLUCOSIDASE 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

3 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	3	MONDO:0002412	MONDO:0002412

2 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
PHASE3	4
Not specified	3
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03911505	PHASE3	ACTIVE_NOT_RECRUITING	ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT04808505	PHASE3	RECRUITING	A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
NCT03729362	PHASE3	COMPLETED	A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
NCT04138277	PHASE3	COMPLETED	A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
NCT02675465	PHASE1/PHASE2	COMPLETED	First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
NCT03865836	Not specified	AVAILABLE	Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
NCT04327973	Not specified	AVAILABLE	Expanded Access for ATB200/AT2221 for the Treatment of IOPD
NCT06121011	Not specified	RECRUITING	A Global Prospective Observational Registry of Patients With Pompe Disease

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).