Spinal muscular atrophy
Neurology· 4 reviewed drugs· 3484 cited findings behind them
What the regulatory reviews cover
Regulatory review documents for several drugs treating spinal muscular atrophy (SMA) are available in this library. These include Evrysdi, indicated for 5q SMA in patients 2 months of age and older with specific clinical diagnoses or SMN2 copies, which showed a median SMN protein increase after 4 weeks of >2 fold. Zolgensma, indicated for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1 or up to 3 copies of the SMN2 gene, demonstrated a median survival of SMNΔ7 mice (ICV, highest dose) of 282 days compared to 17.5 days for untreated mice. Itvisma, also indicated for 5q SMA with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older, similarly showed a median survival of SMNΔ7 mice with the highest ICV dose of AVXS-101 of 282 days. Spinraza, for which an indication is not provided in the source material, showed an improvement in motor milestone development of 40 compared to 0 in the control group.
It is important to note that these drugs were studied in different trials, and therefore, the reported figures are not directly comparable head-to-head.
The drugs — one cited result each
| Evrysdi Roche Holding AG | Median SMN protein increase after 4 weeks: >2 fold Roche Holding AG — Evrysdi EMA EPAR public assessment report, p.82 |
| 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 |
| Spinraza Biogen Inc. | Improvement in motor milestone development: 40 Biogen Inc. — Spinraza FDA Review (NDA209531, ORIG 1, 2016), p.136 |
Different drugs were studied in different trials — these figures are not head-to-head comparisons.
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Lists reflect the regulatory review documents in this library, not all therapies that exist. Educational reference only — not medical or investment advice.