Zolgensma(onasemnogene abeparvovec)
Neurologyยท 747 cited findingsยท 1 source document
Company: Novartis AG โ
What the regulatory reviews say
Zolgensma (onasemnogene abeparvovec) is a gene therapy indicated for the treatment of spinal muscular atrophy (SMA). Without treatment, SMA Type 1 has a life expectancy of less than two years. Nusinersen (Spinraza) was an available therapy for SMA, receiving EMA approval in mid-2017, prior to Zolgensma's marketing authorization application submission on 9 October 2018.
In preclinical studies, Zolgensma demonstrated efficacy in SMNฮ7 mice, with the highest dose (ICV) resulting in a median survival of 282 days, compared to 17.5 days for untreated SMNฮ7 mice. Transduction of motor neurons was observed, with 72% in lumbar motor neurons at the highest dose. Clinical studies showed increases in peroneal CMAP amplitude by 1.67 mV (320%) and ulnar CMAP amplitude by 0.87 mV (125%).
Safety data from clinical trials indicated that 99% of patients experienced at least one treatment-emergent adverse event (TEAE), with 58% of TEAEs related to Zolgensma. At least one serious adverse event (SAE) occurred in 46% of patients, and 40% experienced at least one Grade 3 or higher TEAE. Discontinuations due to fatal TEAEs occurred in 2.7% of patients.
Key figures โ cited to the source page
| Median survival of SMNฮ7 mice (ICV, highest dose) | 282 days | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.29 |
| Median survival of untreated SMNฮ7 mice | 17.5 days | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.29 |
| Peroneal CMAP amplitude increase | 1.67 mV | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.61 |
| Patients experiencing at least 1 TEAE | 99 % | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.104 |
| Patients with at least one SAE | 46 % | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.104 |
| Patients discontinued due to fatal TEAEs | 2.7 % | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.104 |
| SMA Type 1 life expectancy without treatment | less than two years | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.13 |
| Marketing authorization application submission date | 9 October 2018 | Novartis AG โ Zolgensma EMA EPAR public assessment report (E, p.6 |
Source documents
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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials โ values are not head-to-head comparisons). Educational reference only โ not medical or investment advice.