Zolgensma(onasemnogene abeparvovec)
Zolgensma (onasemnogene abeparvovec) is a gene pharmaceutical. Onasemnogene abeparvovec was first approved as Zolgensma on 2019-05-24. It is used to treat spinal muscular atrophy in the USA. It has been approved in Europe to treat spinal muscular atrophy.
Download report
Favorite
Top 200 Pharmaceuticals by Retail Sales
Commercial
Therapeutic Areas
Therapeutic Area | MeSH |
|---|---|
| nervous system diseases | D009422 |
Trade Name
FDA
EMA
Zolgensma
Drug Products
FDA
EMA
Reference product - 351(a)
Reference product - 351(a)
Interchangeable product - 351(k)
Interchangeable product - 351(k)
Biosimilar product - 351(k)
Biosimilar product - 351(k)
Onasemnogene abeparvovec
Tradename | Proper name | Company | Number | Date | Products |
|---|---|---|---|---|---|
| Zolgensma | onasemnogene abeparvovec-xioi | Novartis | A-125694 RX | 2019-05-24 | 1 products |
Labels
FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| zolgensma | Biologic Licensing Application | 2021-03-16 |
Indications
FDA
EMA
Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| spinal muscular atrophy | EFO_1001992 | D009134 | G12.1 |
Agency Specific
FDA
EMA
Expiration | Code | ||
|---|---|---|---|
onasemnogene abeparvovec, Zolgensma, Novartis Gene Therapies, Inc. | |||
| 2031-05-24 | Reference product excl. | ||
| 2026-05-24 | Orphan excl. | ||
Patent Expiration
No data
HCPCS
Code | Description |
|---|---|
| J3399 | Injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15 vector genomes |
Clinical
Clinical Trials
11 clinical trials
View more details
Mock data
Subscribe for the real data
Subscribe for the real data
Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Spinal muscular atrophies of childhood | D014897 | Orphanet_83419 | G12.0 | 1 | — | 1 | 1 | 1 | 4 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Spinal muscular atrophy | D009134 | EFO_0008525 | G12.1 | 1 | — | 3 | — | 2 | 6 |
| Genetic therapy | D015316 | — | — | 1 | — | — | 1 |
Indications Phases 2
No data
Indications Phases 1
No data
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
View more details
Drug
General
| Drug common name | ONASEMNOGENE ABEPARVOVEC |
| INN | onasemnogene abeparvovec |
| Description | Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein.
|
| Classification | Gene |
| Drug class | — |
| Image (chem structure or protein) | |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | 1922968-73-7 |
| RxCUI | — |
| ChEMBL ID | CHEMBL4297240 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | DB15528 |
| UNII ID | MLU3LU3EVV (ChemIDplus, GSRS) |
Target
Agency Approved
No data
Alternate
No data
Variants
Clinical Variant
No data
Financial
Zolgensma - Novartis
$
€
£
₣
Mock data
Subscribe for the real data
Subscribe for the real data
Mock data
Subscribe for the real data
Subscribe for the real data
Tabular view
Trends
PubMed Central
Top Terms for Disease or Syndrome:
Mock data
Subscribe for the real data
Subscribe for the real data
Additional graphs summarizing 1,165 documents
View more details
Safety
Black-box Warning
Black-box warning for: Zolgensma
Adverse Events
Top Adverse Reactions
Mock data
Subscribe for the real data
Subscribe for the real data
48,556 adverse events reported
View more details
Premium feature
Learn more about premium features at pharmakb.com
Learn more