Etranacogene Dezaparvovec

Summary

Etranacogene Dezaparvovec (CHEMBL5095451) is an approved gene (ATC B02BD16); indicated across 2 conditions including hemophilia b.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: B02BD16
• Indications: 2 conditions
• Clinical trials: 4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095451
Name	Etranacogene Dezaparvovec
Type	Gene
Max phase	4
ATC	B02BD16

Also known as: AMT-061, Etranacogene dezaparvovec, Etranacogene dezaparvovec-dlrb, Hemgenix, ETRANACOGENE DEZAPARVOVEC

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

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
hemophilia B	4	MONDO:0010604	MONDO:0010604

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

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
Not specified	2
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03569891	PHASE3	COMPLETED	HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
NCT03489291	PHASE2	COMPLETED	Dose Confirmation Trial of AAV5-hFIXco-Padua
NCT05962398	Not specified	ENROLLING_BY_INVITATION	Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
NCT06008938	Not specified	RECRUITING	An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

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).