Onasemnogene abeparvovec

Phase 3Gene therapy

Onasemnogene abeparvovec is a gene therapy that works as a SMN1 Gene Therapy.[2] It is being investigated across 2 indications, with its most advanced program in Phase 3.[1]

Onasemnogene abeparvovec is one of 27 gene therapy programs targeting Spinal Muscular Atrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Spinal Muscular AtrophyActive, not recruiting

Spinal Muscular Atrophy (SMA)Active, not recruiting

Spinal Muscular AtrophyPhase 3

Active, not recruiting

Spinal Muscular Atrophy (SMA)Phase 3

Active, not recruiting

Competition

Competitive Landscape

35 competitors in Spinal Muscular Atrophy

#DrugCompanyPhaseTrials

Evrysdi

Roche

Approved

8 2

Spinraza

Biogen

Phase 3

20 3

Prilosec

DDexcel Pharma Technologies Ltd.

Amondys 45

Depakote

2 6

Droxia

TTheravia

Phase 2

3 7

Celebrex

PPhiladelphia Eye Associates

Phase 2

1 8

Rilutek

AAzurity Pharmaceuticals

Phase 2

1 9

RO7204239

Roche

Phase 3

1 10

Genotropin

EMD Serono

Phase 2

—

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2030

Active, not recruiting

Long-Term Follow-up

NCT03421977

Q2 2031

Phase 3Active, not recruiting

Spinal Muscular Atrophy (SMA)

NCT05335876

Q2 2032

Post-ApprovalNot yet recruiting

Spinal Muscular Atrophy

NCT07448610

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Completed· Novartis logo

Novartis·Phase 3·NCT03461289·33 enrolled

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Terminated· Novartis logo

Novartis·Phase 1·NCT03381729·32 enrolled

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Completed· Novartis logo

Novartis·Phase 3·NCT03306277·22 enrolled

Show 4 more trials

ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

Not yet recruiting· Novartis logo

Novartis·Phase 4·NCT07448610·36 enrolled

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

Completed· Novartis logo

Novartis·Phase 3·NCT03505099·30 enrolled

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Completed· Novartis logo

Novartis·Phase 3·NCT03837184·2 enrolled

SPR1NT: Onasemnogene Abeparvovec Pre-Symptomatic SMA

Completed· Novartis logo

Novartis·Phase 3·NCT04042025·30 enrolled

Long-term Follow-up1 trial

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Active, not recruiting· Novartis logo

Novartis··NCT03421977·13 enrolled

Spinal Muscular Atrophy (SMA)1 trial

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials

Active, not recruiting· Novartis logo

Novartis·Phase 3·NCT05335876·20 enrolled

Sources

References

[1]
ClinicalTrials.gov — 9 trial records for Onasemnogene abeparvovec. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Onasemnogene abeparvovec compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Onasemnogene abeparvovec. EMBL-EBI & Wellcome Sanger Institute.
[4]
PubMed — 1 publication indexed for Onasemnogene abeparvovec. U.S. National Library of Medicine.
[5]
SEC EDGAR — Novartis company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: SMN1 Gene Therapy[2]
Modality: Gene therapy[2]
Highest Phase: Phase 3[1]
Company: Novartis

Key Publications[4]

One-Time Gene Therapy With Onasemnogene Abeparvovec in Pre-Symptomatic SMA: SPR1NT

N Engl J MedSep 22, 2022DOI: 10.1056/NEJMoa2205091

Onasemnogene abeparvovec

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2030

Q2 2031

Q2 2032

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

Long-term Follow-up1 trial

Spinal Muscular Atrophy (SMA)1 trial

References

Drug Info

Quick Facts

Key Publications[4]

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