Update – 6 November 2026: The U.S. Food and Drug Administration (FDA) has issued a safety communication announcing an investigation into a possible serious risk of malignancy associated with Zolgensma, following reports of tumours in treated children, including two confirmed cases of tumour development directly linked to Zolgensma. FDA has indicated that this risk is relevant to both Zolgensma and Itvisma, while also stating that the benefits of both treatments continue to outweigh their potential risks for their approved uses. FDA is continuing to investigate the risk and assess whether further regulatory action is needed.
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.
