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 14 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 AtrophyRecruiting

Spinal Muscular Atrophy (SMA)Active, not recruiting

Spinal Muscular AtrophyPhase 3

Recruiting

Spinal Muscular Atrophy (SMA)Phase 3

Active, not recruiting

Competition

Competitive Landscape

15 competitors in Spinal Muscular Atrophy

#DrugCompanyPhaseTrials

Evrysdi

Roche

Approved

7 2

Spinraza

Biogen

Phase 3

17 3

Prilosec

DDexcel Pharma Technologies Ltd.

Amondys 45

Salanersen

1 6

Onasemnogene abeparvovec

Novartis

Phase 3

7 7

Apitegromab

SScholar Rock, Inc.

Phase 3

4 8

Taldefgrobep Alfa

BBiohaven Therapeutics Ltd.

Phase 3

1 9

Onasemnogene Abeparvovec-Xioi

NNovartis Gene Therapies

Phase 3

1 10

NMD670

NNMD Pharma A/S

Phase 2

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

Timeline

Expected Milestones

Q4 2030

Active, not recruiting

Spinal Muscular Atrophy 1

NCT03421977

Q2 2031

Phase 3Active, not recruiting

Spinal Muscular Atrophy (SMA)

NCT05335876

Q2 2032

ApprovedRecruiting

Spinal Muscular Atrophy

NCT07448610

Q4 2035

Phase 3Active, not recruiting

Spinal Muscular Atrophy

NCT04042025

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

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

Completed· Novartis logo

Novartis·Phase 3·NCT03306277·22 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 Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Completed· Novartis logo

Novartis·Phase 3·NCT03461289·33 enrolled

Show 4 more trials

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

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

Terminated· Novartis logo

Novartis·Phase 1·NCT03381729·32 enrolled

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

Recruiting· Novartis logo

Novartis·Approved·NCT07448610·36 enrolled

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101

Active, not recruiting· Novartis logo

Novartis·Phase 3·NCT04042025·85 enrolled

Spinal Muscular Atrophy 11 trial

A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 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 — 10 publications 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]

Predicting Intolerance to Deep-Inspiration Breath-Hold for Abdominal Cancer Radiation Therapy.

Adv Radiat OncolDec 1, 2026DOI: 10.1016/j.adro.2026.102165

Metabolic reprogramming in diabetic panvascular disease: Molecular mechanism and therapeutic strategies.

iScienceOct 16, 2026DOI: 10.1016/j.isci.2026.117578

A biomaterials roadmap from retinoic acid biology to kidney regenerative therapy.

iScienceOct 16, 2026DOI: 10.1016/j.isci.2026.117554

Diet and nutrition in chronic heart failure: A scientific statement of the Polish Society of Dietetics.

Adv Clin Exp MedOct 2, 2026DOI: 10.17219/acem/225017

Eating Disorders in Transgender, Non-Binary, and Gender Diverse Populations: A Meta-Synthesis of Qualitative Findings.

Int J Eat DisordOct 2, 2026DOI: 10.1002/eat.70228

Identification of glycoproteins as candidate biomarkers for nasopharyngeal carcinoma using Fe(3)O(4)@PDA@MOF-IDA and mM-TiO(2)@Cys enrichment and HPLC-MS proteomic analysis.

Anal MethodsOct 2, 2026DOI: 10.1039/d6ay01497f

Diet and nutrition in obesity treated with incretin-based therapies: A scientific statement of the Polish Society of Dietetics.

Adv Clin Exp MedOct 2, 2026DOI: 10.17219/acem/235937

Living With ALK Fusion-Positive NSCLC: A French Nationwide Survey of the Psychosocial Impact Among Patients, Caregivers, and Health Care Professionals.

JTO Clin Res RepOct 1, 2026DOI: 10.1016/j.jtocrr.2026.101038

Duodenal Obstruction and Grade III Acute Cholangitis Following Pancreaticoduodenal Artery Aneurysm Rupture During Conservative Management of Acute Type B Aortic Dissection: A Case Report.

CureusAug 1, 2026DOI: 10.7759/cureus.115546

Emerging Neuromodulation, Electrical Stimulation, and Cellular Therapies for Adult Cervical Spinal Cord Injuries in the United States: A Scoping Review.

CureusAug 1, 2026DOI: 10.7759/cureus.115549

Onasemnogene abeparvovec

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2030

Q2 2031

Q2 2032

Q4 2035

Trials by Indication[1]

Spinal Muscular Atrophy7 trials

Spinal Muscular Atrophy 11 trial

Spinal Muscular Atrophy (SMA)1 trial

References

Drug Info

Quick Facts

Key Publications[4]

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