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Onasemnogene Abeparvovec-xioi
Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy.
Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy. Used for Spinal muscular atrophy (SMA) in pediatric patients.
At a glance
| Generic name | Onasemnogene Abeparvovec-xioi |
|---|---|
| Also known as | Zolgensma, AVXS-101, OAV101 |
| Sponsor | Novartis Gene Therapies |
| Drug class | Gene therapy |
| Target | SMN1 gene |
| Modality | Biologic |
| Therapeutic area | Neurology |
| Phase | FDA-approved |
Mechanism of action
The drug uses an AAV9 vector to cross the blood-brain barrier and deliver the SMN1 gene directly to motor neurons and other tissues. Once delivered, the functional SMN1 gene is expressed, producing survival motor neuron (SMN) protein, which is deficient or absent in spinal muscular atrophy patients due to mutations in the endogenous SMN1 gene. This protein restoration halts or reverses the progressive motor neuron degeneration characteristic of the disease.
Approved indications
- Spinal muscular atrophy (SMA) in pediatric patients
Common side effects
- Elevated transaminases (ALT/AST)
- Thrombocytopenia
- Hepatotoxicity
- Fever
Key clinical trials
- A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy (PHASE2)
- Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies (PHASE3)
- Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 (PHASE3)
- Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (PHASE3)
- Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 (PHASE3)
- Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (PHASE1)
- Long-Term Follow-up Study for Patients From AVXS-101-CL-101
- Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Onasemnogene Abeparvovec-xioi CI brief — competitive landscape report
- Onasemnogene Abeparvovec-xioi updates RSS · CI watch RSS
- Novartis Gene Therapies portfolio CI