STAT+: Novartis cites cases of previously unknown tumors possibly tied to its SMA gene therapy

STAT PlusPharmalot Novartis cites cases of previously unknown tumors possibly tied to its SMA gene therapy Disclosure raises concerns among families, physicians, and advocacy groups Manage alerts for this article Save this story for later Share this article By Ed Silverman and Jason MastNov. 5, 2026 Ed Silverman[email protected]Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease.

Confidential tips can be sent on Signal at JasonMast.77. Novartis has acknowledged seven previously unknown instances in which tumors were found in children who were treated with its spinal muscular atrophy therapy, a surprising development that has raised concerns among families, physicians, and patient advocacy groups. The cases, which for the moment are being seen as only a potential safety signal, were publicized on Thursday by an advocacy group and on a Facebook page after Novartis earlier this week disclosed the information to neurologists.

One advocacy group, SMA Europe, issued a statement saying that regulatory authorities are now examining the cases. Advertisement The drugmaker issued its own statement saying that since its treatment — called Zolgensma — was approved in 2019, it has received nine reports of tumors in previously treated patients. Of those, two cases to date showed evidence that the tumors may be linked to the treatment itself.

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Subscribe Log In biotechnology, Gene therapy, Novartis, Pharmaceuticals, rare diseases, STAT+ Submit a correction requestReprints Ed Silverman Pharmalot Columnist, Senior Writer Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter. Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease.

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