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onasemnogene abeparvovec

Itvisma, Zolgensma · ATC code M09AX09

Also known as: Onasemnogen abeparvovec

Key facts
Active substance
onasemnogene abeparvovec
ATC code
M09AX09
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
Itvisma 1.2 × 1014 vector genomes solution for injection · Zolgensma 2 × 1013 vector genomes/mL solution for infusion
Marketing authorisation holder
Novartis Europharm Limited
Information as of
2026-10-27

Key facts

Onasemnogene abeparvovec is an AAV9-based gene therapy that delivers a functional SMN1 gene copy. It is given once for 5q spinal muscular atrophy (SMA): Zolgensma by infusion, Itvisma intrathecally.

What it is used for

  • Zolgensma: 5q SMA with a bi-allelic SMN1 mutation and a clinical diagnosis of SMA Type 1, or up to 3 copies of the SMN2 gene
  • Itvisma: 5q SMA with a bi-allelic SMN1 mutation in patients 2 years of age and older

Dosing

  • Zolgensma: single intravenous infusion, dose based on body weight (table in the full text)
  • Itvisma: single intrathecal injection at a fixed dose; not for patients previously treated with onasemnogene abeparvovec
  • Beforehand: AAV9 antibody test, liver function, creatinine, blood count (for Zolgensma also troponin-I)
  • Corticosteroid cover: prednisolone 1 mg/kg/day from 24 hours before and for 30 days, then gradual taper depending on liver tests
  • Only in specialised centres; postpone if acute or uncontrolled infection

Contraindications

  • Hypersensitivity to the active substance or any excipient

Important warnings

  • Hepatotoxicity (raised ALT/AST); corticosteroids before and after treatment and liver monitoring
  • Thrombocytopenia, typically within the first week; monitor platelets at least weekly for the first month
  • Thrombotic microangiopathy, potentially life-threatening
  • Serious immune response possible; patients should be clinically stable and free of infection
  • Elevated anti-AAV9 antibody titres: safety and efficacy not established

Side effects

  • Zolgensma: most frequent hepatic enzyme increased (24.2%), hepatotoxicity (9.1%), vomiting (8.1%), thrombocytopenia (6.1%), troponin increased (5.1%), pyrexia (5.1%)
  • Itvisma: most frequent upper respiratory tract infection (41.7%), pyrexia (36.2%), vomiting (28.3%), headache (13.4%), increased hepatic enzyme (9.4%)
  • Rare anaphylactic reactions; uncommon thrombotic microangiopathy

Interactions

  • No interaction studies; limited experience with hepatotoxic substances
  • Adjust vaccination schedule for corticosteroid use; seasonal RSV prophylaxis recommended; no live vaccines (e.g. MMR, varicella) on immunosuppressive steroid doses

Pregnancy and breastfeeding

  • Pregnancy: no data; treat only after a thorough benefit-risk evaluation
  • Breast-feeding: no data; decide on breast-feeding weighing benefit and risk

Summary of the official product information, written with AI (Claude, Anthropic) on 2026-10-28. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

Common questions – ask Medelix

For patients

Package leaflet, verbatim

Package leaflet for patients →

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

  • ItvismaNovartis Europharm Limited · EMEA/H/C/006498
  • ZolgensmaNovartis Europharm Limited · EMEA/H/C/004750

Source: European Medicines Agency (EMA), product information

Brand names in 3 more European countries
Iceland
Itvisma, Zolgensma
Liechtenstein
Itvisma, Zolgensma
Norway
Itvisma, Zolgensma

EMA – Article 57 product data (EU/EEA), 2026-10-28

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

Other substances in the same group

Another question about this substance?

Medelix answers clinical questions from guidelines, studies and product information – every claim with its source.

Sources

  1. European Medicines Agency. Itvisma – EPAR, Product Information (OF THE TEXT Detailed information on this medicinal product i). 2026-10-27. https://www.ema.europa.eu/en/documents/product-information/itvisma-epar-product-information_en.pdf
  2. European Medicines Agency. Zolgensma – EPAR, Product Information (OF THE TEXT Detailed information on this medicinal product i). 2026-10-27. https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf

Editorial: Medelix editorial team · last updated 2026-10-27 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.