Zolgensma (onasemnogene abeparvovec-xioi) · Novartis
ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ] . The use of ZOLGENSMA in patients with advanced SMA (e.g.
Details
- Status
- Prescription
- First Approved
- 2019-05-23
- Patent Cliff
- 2031
- Revenue
- $307M (Q4-2025)
- Routes
- Intravenous
- Dosage Forms
- Injection
Zolgensma Approval History
What Zolgensma Treats
1 FDA approvalsOriginally approved for its first indication in 2019 .
- Other (1)
Other
(1 approval)- • Approved indication (May 2019)
Zolgensma Boxed Warning
SERIOUS LIVER INJURY and ACUTE LIVER FAILURE Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )]. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing. Administer systemic corticosteroid to all patien...
WARNING: SERIOUS LIVER INJURY and ACUTE LIVER FAILURE Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )]. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing. Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated [see Dosage and Administration ( 2.1 , 2.3 )]. WARNING: SERIOUS LIVER INJURY and ACUTE LIVER FAILURE See full prescribing information for complete boxed warning. Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA. ( 5.1 ) Patients with preexisting liver impairment may be at higher risk. ( 5.1 ) Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing. Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated. ( 2.1 , 2.3 )
Zolgensma Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Novartis's other novel FDA approvals
Other CDER-designated drugs from the same sponsor (2016–2025).
Clinical Trial Registry
6 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03381729 STRONG results posted | AVXS-101-CL-102 2020-003678-28, COAV101A12102 | Ph 1 | terminated | Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy |
| NCT03306277 STR1VE results posted | AVXS-101-CL-303 2020-000095-38, COAV101A12302 | Ph 3 | completed | Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 |
| NCT03837184 results posted | AVXS-101-CL-306 194664, 201900208 | Ph 3 | completed | Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies |
| NCT03505099 SPR1NT results posted | AVXS-101-CL-304 2017-004087-35, JapicCTI-184203 | Ph 3 | completed | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 |
| NCT03461289 STRIVE-EU results posted | AVXS-101-CL-302 2017-000266-29, COAV101A12301 | Ph 3 | completed | Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 |
| NCT04042025 | AVXS-101-LT-002 2019-002611-26, 205305 | Ph 3 | active not recruiting | Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
Zolgensma FDA Label Details
Indications & Usage
ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies ] . ZOLGENSMA is an adeno-associated virus (AAV...
WARNING: SERIOUS LIVER INJURY and ACUTE LIVER FAILURE Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be...
Pro Intelligence Preview
Deep insights for Zolgensma
Revenue Insights
- • Q4-2025: $307M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2031
- • Generic/biosimilar risk
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.