Novartis has confirmed nine reports of tumors in children treated with its spinal muscular atrophy gene therapy Zolgensma® (onasemnogene abeparvovec-xioi). Seven of these cases are newly disclosed. Two cases have shown evidence of vector integration into the patients’ DNA, and the patients are being monitored.
Novartis stated that patient safety is a priority and that it has notified health authorities about these cases. To date, Zolgensma has treated over 6,000 patients worldwide. The potential risk of tumors due to vector integration is included in Zolgensma’s prescribing information.
The Muscular Dystrophy Association (MDA) and SMA Europe have both expressed the need for transparent communication regarding these reports. MDA is reviewing the available data with clinical experts and has reached out to Novartis for more information.
Novartis’ Zolgensma Group generated $1.071 billion in net sales during the first three quarters of this year, reflecting a 16% increase from the same period in 2025. The company’s shares saw a slight dip following the announcement.
Source: genengnews.com · Image: NHGRI-85172 by Darryl Leja, NHGRI, Public domain, via Wikimedia Commons
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