Zolgensma (IV Onasemnogene Abeparvovec)
Zolgensma (IV Onasemnogene Abeparvovec)
Page last updated: 4th November 2026
Zolgensma is a gene therapy. It delivers a healthy copy of the SMN1 gene to motor neurons. The gene is packaged inside an inactive shell of a virus called AAV9 (adeno-associated virus 9). This is so that it can be transported to the target cells.
The National Multidisciplinary Team (MDT) for Zolgensma®, which has coordinated access to onasemnogene abeparvovec, (brand name Zolgensma®), for children with SMA in England over the past five years, would like to share an update about new safety information recently communicated by Novartis, the company that makes Zolgensma®.
Novartis has informed healthcare professionals of 9 reports worldwide of tumours diagnosed in children who had previously received Zolgensma®. The information available about these children is currently limited. So far, pathology testing has been completed on the tumours of 3 of the 9 children. This has shown that 2 of these tumours are specifically related to Zolgensma® treatment, while 1 appears to be unrelated. Further information is awaited from the regulatory authorities.
We understand that this news may be worrying for families, and we are sharing it with you in the interests of openness and transparency. It is important to keep it in perspective. More than 6,000 children worldwide have received Zolgensma®, and the number of reported and confirmed cases is small.
Based on the information currently available, no changes to your child’s routine care or follow-up are recommended, and no additional scans, tests or monitoring are needed at this time.
The National MDT for Zolgensma® is reviewing all available information and will continue to monitor the situation closely. We are committed to keeping families, healthcare professionals and patient organisations informed as our understanding develops, and we will share further information as soon as it becomes available.
If you have any questions or concerns about your child, please contact your child’s neuromuscular team, who will be happy to talk things through with you.
On the 5th November 2026 Novartis provided SMA Europe with the following statement for sharing with the community:
“More than 6,000 patients have been treated with Zolgensma® across clinical trials, managed access programs and commercial treatment. As part of Novartis’ ongoing commitment to monitoring the safety of our medicines, we continually evaluate new safety information and assess all reported cases.
Since first approval in 2019, Novartis has received 9 reports of tumors in patients previously treated with Zolgensma. Of those, two cases to date have shown evidence of vector DNA integration into the DNA of the patients. In both cases, the patients underwent treatment and continue to be monitored. The potential risk of tumors due to vector integration is currently included in the Zolgensma prescribing information.
Patient safety is our priority. In each case, we timely and appropriately notified health authorities and will continue to work with them on next steps. We appreciate the opportunity to respond to community questions.”
Researchers have tested intravenous (IV) Zolgensma in human clinical trials with children up to the age of 2 years since 2014. Zolgensma is not a cure for 5q SMA but in these trials, it has been consistently shown to have an acceptable safety profile and to be well-tolerated. When compared to no treatment, it has led to clinically meaningful improvements in muscle function. Trials with older heavier children are ongoing. These include giving Zolgensma via a lumber puncture (intrathecal (IT) administration).
We have worked hard with the SMA Community, MDUK, TreatSMA and clinicians, to advocate for access.