Recent Zolgensma safety concerns: what Novartis has reported

SMA Europe has issued information on recent Zolgensma safety concerns after reports of tumours in patients who had previously received treatment. The organisation says families need clear and reliable information, and should not have to piece the issue together from social media, rumours or incomplete reports.

The figures behind recent Zolgensma safety concerns

Novartis says more than 6,000 patients have been treated with Zolgensma across clinical trials, managed access programmes and commercial treatment. Since first approval in 2019, the company has received nine reports of tumours in patients previously treated. Two of those cases to date show evidence of vector DNA integration into the patients’ DNA. Both underwent treatment and continue to be monitored. Novartis also states that the potential risk of tumours from vector integration is already included in the prescribing information, and that it notified health authorities in each case.

Why it matters

The nine reports are set against more than 6,000 treated patients, a denominator Novartis gives but does not turn into a rate. What remains unclear is what the two integration cases mean for people already treated, and whether extra monitoring is recommended. SMA Europe says the SMA community now needs timely public communication. It has pressed Novartis on exactly this and is collecting questions from families and member organisations to put to the company.

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SMA Europe says the relevant regulators are assessing the evidence. For another recent example, see the EMA’s safety review of Deanxit.

The UK update, relayed from SMA UK after information from the UK National Multidisciplinary Team for Zolgensma, contains clinical recommendations for the UK context. Families with individual concerns should speak to their treating healthcare team.

Source: SMA Europe, 5 November 2026, quoting a Novartis statement.