Information regarding recent Zolgensma safety concerns
SMA Europe is aware of information currently circulating regarding Zolgensma and reports of tumours in children who have previously received treatment.
We know that this information may be deeply concerning for families, particularly for parents whose children have already received Zolgensma or who are currently considering treatment. Families need clear, reliable and understandable information and should not be left to piece together a potential safety concern from social media, rumours or incomplete reports.
We understand that the relevant regulatory authorities are actively involved and are currently assessing the available evidence and determining what further steps may be needed. We trust in the regulatory processes established to evaluate safety signals and to take the necessary measures to protect patients and ensure the continued safety of medicines on the market.
SMA Europe has also been informed about the situation through confidential channels and have made clear to Novartis that the SMA community now needs timely public communication. We have strongly urged the company to provide clear information on what is currently known, what remains uncertain, what this may mean for people who have already received treatment, and whether any additional monitoring or action is recommended.
Today 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."
For the time being, further publicly available information has been provided by SMA UK following information from the UK National Multidisciplinary Team for Zolgensma. This can be found here: SMA UK – Zolgensma. Please note that the clinical recommendations in this update relate to the UK context. Families with individual concerns should speak with their treating healthcare team.
SMA Europe is collecting questions from families and our member organisations and will raise these directly with Novartis. If there are questions you believe need to be answered, please share them with us.
Our priority is to support the SMA community through this situation, to advocate for transparent and timely communication, and to ensure that the concerns and questions of patients and families are heard. We will share further reliable information as soon as it becomes available.