TheTechno Optimist.

Del-zota (delpacibart zotadirsen)Novartis—FDA accepts BLA for del-zota in DMD44 with priority reviewLeqvioNovartis—Leqvio (inclisiran) Q2 2026 revenue reaches $480M, up 59% year-over-year, with peak sales target of $4-5BItvisma (onasemnogene abeparvovec-brve)Novartis—EU approves Itvisma for SMA in children 2+ and adultsLeqvioNovartis—FDA updates Leqvio label to recognize cardiovascular outcomes benefits

Novartis

Gene therapy, rare diseases, oncology

CHpublic~78,759 staffest. 1996novartis.com (opens novartis.com)Checked 11 Oct

The brief

Analyst view
  • Itvisma approved for SMA in children 2+ and adults.
  • Del-zota (AOC) for DMD44 has BLA accepted with priority review.
  • Acquired Avidity for $12B in 2024 to expand neuromuscular pipeline.

Technical approach

As reported
AAV gene therapy

One-time genetic fix

AAV-mediated gene replacement targeting genetic root causes.

AOC therapy

Antibody-delivered antisense

Antibodies target muscle cells delivering splice-modifying antisense.

Rare diseases

Neuromuscular focus

Concentrate on severe inherited neuromuscular disease with unmet needs.

Primer