Onasemnogene Abeparvovec

Summary

Onasemnogene Abeparvovec (CHEMBL4297240) is an approved gene (ATC M09AX09); indicated across 5 conditions including spinal muscular atrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: M09AX09
• Indications: 5 conditions
• Clinical trials: 5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297240
Name	Onasemnogene Abeparvovec
Type	Gene
Max phase	4
ATC	M09AX09

Also known as: AVXS-101, Onasemnogen abeparvovec, Onasemnogene abeparvovec, Zolgensma, ONASEMNOGENE ABEPARVOVEC

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
spinal muscular atrophy	4	MONDO:0001516	EFO:0008525

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

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
Not specified	3
PHASE3	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05335876	PHASE3	RECRUITING	Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
NCT02122952	PHASE1	COMPLETED	Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
NCT04174157	Not specified	RECRUITING	Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
NCT06019637	Not specified	RECRUITING	A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
NCT03955679	Not specified	APPROVED_FOR_MARKETING	AveXis Managed Access Program Cohort for Access to AVXS-101

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).