Idursulfase

Summary

Idursulfase (CHEMBL1201826) is an approved enzyme (ATC A16AB09); indicated across 2 conditions including mucopolysaccharidosis type 2.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Enzyme
• ATC class: A16AB09
• Indications: 2 conditions
• Clinical trials: 13

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201826
Name	Idursulfase
Type	Enzyme
Max phase	4
ATC	A16AB09

Also known as: DRX006A, EC 3.1.6.13, EC-3.1.6.13, Elaprase, Gc-1111, GC1111, HGT-2310, HGT2310, Idursulfasa, Idursulfase, Sulfatase, l-idurono-

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

2 indications (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
mucopolysaccharidosis type 2	4	MONDO:0010674	MONDO:0010674
mucopolysaccharidosis	2	MONDO:0019249	MONDO:0019249

Clinical trials

Total trials: 13.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00607386	PHASE4	COMPLETED	Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
NCT05058391	PHASE4	COMPLETED	A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
NCT05494593	PHASE4	WITHDRAWN	A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
NCT04573023	PHASE3	ACTIVE_NOT_RECRUITING	A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
NCT05371613	PHASE2/PHASE3	RECRUITING	A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
NCT06031259	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
NCT00630747	PHASE2/PHASE3	COMPLETED	Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
NCT02412787	PHASE2/PHASE3	COMPLETED	Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
NCT03920540	PHASE3	COMPLETED	A Study of GC1111 in Hunter Syndrom Patients
NCT01506141	PHASE1/PHASE2	COMPLETED	An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
NCT02663024	PHASE2	UNKNOWN	Study of Idursulfase-beta (GC1111) in Hunter Syndrome
NCT00882921	Not specified	COMPLETED	An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
NCT01602601	Not specified	COMPLETED	A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

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).