Novartis reports that nine children treated with its gene therapy Zolgensma for spinal muscular atrophy developed tumors, with two cases showing evidence that the viral vector fused into patient DNA. Over 6,000 patients have received the therapy since 2019. Patient advocacy groups SMA Europe and Cure SMA have urged Novartis to provide transparent safety data, as medical experts note these findings confirm long-feared risks associated with adeno-associated virus delivery tools.
Novartis reports Zolgensma tumor cases
- ▪In two of the nine tumor cases in spinal muscular atrophy patients, doctors found evidence that the viral vector used in Zolgensma inserted its genetic material into the patients' DNA
- ▪Novartis stated that it notified health authorities about the Zolgensma tumor cases and that the potential tumor risk is included in prescribing information
- ▪Novartis reported that nine patients treated with its gene therapy Zolgensma for spinal muscular atrophy subsequently developed tumors
Zolgensma mechanism and prescribing warnings
- ▪The theoretical risk of adeno-associated virus (AAV) integration into patient DNA is listed among warnings in Zolgensma's prescribing information
- ▪Zolgensma uses an adeno-associated virus (AAV) to deliver a gene encoding the SMN protein to treat spinal muscular atrophy in young children
Responses from patient groups and experts
- ▪Patient group SMA Europe called on Novartis to provide clear information on Zolgensma safety concerns rather than leaving families to rely on rumors
- ▪University of Rochester neurologist Sam Mackenzie stated that confirmed adeno-associated virus risks will change treatment calculations for spinal muscular atrophy patients
- ▪U.S. patient group Cure SMA stated that three of nine Zolgensma tumor cases were tested, with one case appearing unrelated to the therapy
Zolgensma reach and sales
- ▪Novartis confirmed that more than 6,000 children have been treated with Zolgensma since gaining initial approval in 2019 for spinal muscular atrophy
- ▪Zolgensma generated $1.1 billion in revenue through the first nine months of 2026, making it the most commercially successful gene replacement therapy
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