Cerliponase Alfa

Summary

Cerliponase Alfa (CHEMBL3544921) is an approved enzyme (ATC A16AB17); indicated across 5 conditions.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB17
• Indications: 5 conditions
• Clinical trials: 4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3544921
Name	Cerliponase Alfa
Type	Enzyme
Max phase	4
ATC	A16AB17

Also known as: BMN 190, BMN-190, Brineura, Cerliponasa alfa, Cerliponase alfa, Immature tripeptidyl-peptidase i, CERLIPONASE 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

5 indication records carry no mapped disease name (EFO/MeSH-only); none shown.

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE1/PHASE2	3
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05152914	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
NCT01907087	PHASE1/PHASE2	COMPLETED	A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
NCT02485899	PHASE1/PHASE2	COMPLETED	An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT04476862	Not specified	ACTIVE_NOT_RECRUITING	Cerliponase Alfa Observational Study in the US

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).