Novartis Discloses New Tumor Cases in Children Treated With Its Gene Therapy
The drugmaker told neurologists this week that nine patients treated with its spinal muscular atrophy therapy Zolgensma have developed tumors since 2019, with two cases showing a possible link to the treatment.
Novartis has disclosed new safety concerns around Zolgensma, its gene therapy for spinal muscular atrophy, after acknowledging seven previously unreported cases of tumors in children who received the treatment. The company said it has now received nine total reports of tumors in treated patients since Zolgensma's 2019 approval, two of which show evidence that may link them to the therapy itself.
Novartis first briefed neurologists on the cases earlier in the week, before an advocacy group and a patient Facebook page made the information public. SMA Europe said regulatory authorities are now examining the reports, and the company has characterized the findings as a potential safety signal rather than a confirmed causal link, pending further review.
Zolgensma remains one of the most high-profile gene therapies on the market, a one-time treatment aimed at a rare and often fatal childhood disease. A safety signal in a therapy this visible rarely stays contained to a medical journal footnote; it becomes a test of how much trust a company built on a single-dose miracle can spend before parents and regulators start asking harder questions.
Source: STAT News
Kyle Duford, Founder & Editor, BRNDWIRE. More from Kyle →
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