Overview
- The FDA cleared Itvisma (onasemnogene abeparvovec‑brve) for patients aged 2 years and older with a confirmed SMN1 mutation.
- In a randomized phase 3 trial of 126 participants aged 2–18 who could sit but had never walked, treated patients improved about 2.4 points on HFMSE over 52 weeks versus 0.5 with sham.
- Itvisma is delivered as a single, fixed‑dose intrathecal injection that does not require adjustment for age or body weight.
- Investigators reported generally similar and manageable adverse events across groups, with common events including upper respiratory tract infection, pyrexia, and vomiting, and effects sustained through one year.
- The therapy, developed by Novartis, offers a potential alternative to lifelong repeat‑dose options such as Spinraza and Evrysdi, with longer‑term durability and safety monitoring still needed.