Laronidase

Summary

Laronidase (CHEMBL1201595) is an approved enzyme (ATC A16AB05); indicated across 6 conditions including mucopolysaccharidosis and glycogen storage disease iii.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB05
• Indications: 6 conditions
• Clinical trials: 12

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201595
Name	Laronidase
Type	Enzyme
Max phase	4
ATC	A16AB05

Also known as: A-l-iduronidase, Aldurazyme, Alronidase, Ec 3.2.1.76, Laronidasa, Laronidase, LARONIDASE

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

6 indications (5 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
mucopolysaccharidosis	4	MONDO:0019249	MONDO:0019249
glycogen storage disease III	1	MONDO:0009291	MONDO:0009291

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

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE1	4
PHASE4	3
PHASE3	2
Not specified	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00144768	PHASE4	COMPLETED	A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
NCT00418821	PHASE4	TERMINATED	A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
NCT05134571	PHASE4	COMPLETED	China Post-marketing Surveillance (PMS) Study of Aldurazyme®
NCT00146770	PHASE3	COMPLETED	Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
NCT06406153	PHASE3	COMPLETED	Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
NCT00741338	PHASE1/PHASE2	COMPLETED	Immune Tolerance Study With Aldurazyme® (Laronidase)
NCT04532047	PHASE1	RECRUITING	PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
NCT00215527	PHASE1	TERMINATED	Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
NCT00786968	PHASE1	TERMINATED	Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
NCT01173016	PHASE1	COMPLETED	Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
NCT00852358	Not specified	COMPLETED	A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
NCT01572636	Not specified	TERMINATED	Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

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).