Valoctocogene Roxaparvovec

Summary

Valoctocogene Roxaparvovec (CHEMBL4297579) is an approved gene (ATC B02BD15); indicated across 2 conditions including hemophilia a.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: B02BD15
• Indications: 2 conditions
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297579
Name	Valoctocogene Roxaparvovec
Type	Gene
Max phase	4
ATC	B02BD15

Also known as: BMN-270, BMN270, Roctavian, Valoctocogen roxaparvovec, Valoctocogene roxaparvovec, VALOCTOCOGENE ROXAPARVOVEC

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 (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
hemophilia A	4	MONDO:0010602	MONDO:0010602

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

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	4
PHASE1/PHASE2	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06224907	PHASE3	ACTIVE_NOT_RECRUITING	Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT03370913	PHASE3	COMPLETED	Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
NCT03392974	PHASE3	COMPLETED	Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT04323098	PHASE3	COMPLETED	Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
NCT04684940	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT02576795	PHASE1/PHASE2	COMPLETED	Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT03520712	PHASE1/PHASE2	TERMINATED	Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

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).