Gene therapies
Neurology· 2 reviewed drugs· 1467 cited findings behind them
What the regulatory reviews cover
This section provides an overview of gene therapies that have undergone regulatory review. Zolgensma is indicated for 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. 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.
Regulatory documents report various outcomes for these therapies. For Zolgensma, the median survival of SMNΔ7 mice (ICV, highest dose) was 282 days, compared to 17.5 days for untreated SMNΔ7 mice. Itvisma showed a median survival of SMNΔ7 mice with the highest ICV dose of AVXS-101 at 282 days, compared to 17.5 days for untreated SMNΔ7 mice. It is important to note that different drugs were studied in different trials, and therefore, the reported figures are not directly comparable head-to-head.
The drugs — one cited result each
| Zolgensma Novartis AG | Median survival of SMNΔ7 mice (ICV, highest dose): 282 days Novartis AG — Zolgensma EMA EPAR public assessment report (E, p.29 |
| Itvisma Novartis AG | Median survival of SMNΔ7 mice with highest ICV dose of AVXS-101: 282 Novartis AG — Itvisma EMA EPAR public assessment report (EME, p.28 |
Different drugs were studied in different trials — these figures are not head-to-head comparisons.
Explore the full evidence
Every finding behind these drugs — searchable, comparable and answerable in plain language — is in the Neurology research library →
Lists reflect the regulatory review documents in this library, not all therapies that exist. Educational reference only — not medical or investment advice.