Novartis reports nine tumor cases possibly linked to Zolgensma gene therapy

Novartis confirmed on November 5, 2026 that it has received nine reports of tumors in children treated with its spinal muscular atrophy gene therapy Zolgensma since its approval in 2019, including seven previously unknown cases. Two cases showed evidence of a direct link to the treatment. Regulatory authorities are examining the cases as a potential safety signal after advocacy group SMA Europe publicized the reports following Novartis' disclosure to neurologists.

Novartis reports tumor cases linked to Zolgensma

• ▪Novartis stated that two of nine reported tumor cases showed evidence that the tumors may be linked directly to its Zolgensma gene therapy
• ▪Novartis confirmed on November 5, 2026 that it has received nine reports of tumors in spinal muscular atrophy patients treated with its gene therapy Zolgensma since its approval in 2019
• ▪Novartis acknowledged seven previously unknown instances in which tumors were found in children who were treated with its spinal muscular atrophy therapy Zolgensma

Disclosure triggers concern among advocacy groups

• ▪Reports of tumors in children treated with Zolgensma raised concerns among families, physicians, and patient advocacy groups
• ▪Advocacy group SMA Europe and a Facebook page publicized the Zolgensma tumor reports on November 5, 2026 after Novartis disclosed the information to neurologists