Onasemnogene abeparvovec
Approved · EMAAlso known as Itvisma, Zolgensma.
Recent clinical, regulatory, research and industry developments relating to this drug.
CHMP positive opinion: Itvisma (EMA)
Profile
Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.
Pharmacology & targets
Known molecular targets and mechanisms supported by curated pharmacology databases.
Regulatory timeline
The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.
Indication: Itvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older.
Evidence ↗Indication: Treatment of 5q spinal muscular atrophy (SMA)
Evidence ↗Indication: Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or… Show full indicationShow less
Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.
Clinical trials
The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.
Late-stage studies
Phase III+ trials still open or recently active — where late-stage evidence is being generated.
Research activity
Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.
Major research themes6
Journals, researchers & institutions
- Journal of hepatology1
- Chand D1
- Kaufmann P1
- Kleyn A1
- Kullak-Ublick G1
- McMillan H1
- Mohr F1
- Montgomery K1
- Sun R1
- Children's Hospital of Eastern Ontario1
- Switzerland; Department of Clinical Pharmacology and Toxicology1
- Washington University School of Medicine1
Related drugs
Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).
Studied across 2 of the same disease areas as Onasemnogene abeparvovec in the shared literature.
Studied across 2 of the same disease areas as Onasemnogene abeparvovec in the shared literature.
- RxNorm (U.S. National Library of Medicine) — drug identity
- ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
- Europe PMC — research literature
- ClinicalTrials.gov — clinical trials
- Regulatory event sources are credited in the Regulatory Timeline above.