Novartis AG — Itvisma EMA EPAR public assessment report (EMEA/H/C/006498, 2026)
Novartis AG · 2026 · EMA EPAR public assessment report · company profile →
Report summary
The EMA/CHMP report provides a positive opinion for Itvisma (onasemnogene abeparvovec), a gene therapy for 5q spinal muscular atrophy (SMA), for patients aged 2 years and older. Itvisma, designated an orphan medicinal product, is not considered similar to Spinraza. The report details Itvisma's manufacturing, stability (24-month shelf life), and quality control. Non-clinical studies in animal models showed dose-response and biodistribution, but also dorsal root ganglia (DRG) toxicity at the lowest tested dose in non-human primates, preventing a NOAEL establishment. Concerns about germline transmission were raised but deemed low risk. Clinical trials demonstrated efficacy, with Study B12301 showing significant improvement in HFMSE scores compared to sham in SMA type 2 patients aged 2-18 years. Study CL-102 also showed improvements in younger patients. The therapeutic indication is for SMA types 2, 3, and 4 in patients aged 2 years and older, excluding SMA type 1 and younger patients due to insufficient data. Safety concerns, consistent with Zolgensma, include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, and DRG toxicity/peripheral sensory neuropathy. Routine and additional risk minimization measures are outlined. Long-term safety data is still being collected.
Key findings — cited to the page
| Final post baseline platelet values (grade 1) | 9.3 % | OAV101B · p.116 |
| Grade 3 decreases in platelet count | 2.0 % | Sham · p.116 |
| Grade 3 decreases in platelet count | 2.7 % | OAV101B · p.116 |
| Grade 4 decreases in platelet count | 2.0 % | Sham · p.116 |
| Final post baseline platelet values (grade 1) | 3.9 % | Sham · p.116 |
| Grade 4 decreases in platelet count | 1.5 % | OAV101B · p.116 |
| Shift to grade 1 decrease in platelet count | 45.3 % | OAV101B · p.116 |
| Shift to grade 1 decrease in platelet count | 23.5 % | Sham · p.116 |
| Indication for Itvisma | treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 6 months of age and older | Itvisma (Onasemnogene abeparvovec) · p.9 |
| Anti-SMN antibodies | 2 subjects | OAV101B · p.52 |
| Median survival of SMNΔ7 mice with 2nd highest ICV dose of AVXS-101 | 274 days | · p.28 |
| Median survival of SMNΔ7 mice with 3rd highest ICV dose of AVXS-101 | 165 days | · p.28 |
Source: Novartis AG ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →