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Zolgensma tumor reports

Novartis reports tumors in children given Zolgensma

The company said two of nine tumor reports since 2019 show a possible link to the gene therapy; regulators are reviewing the cases.

By Theo Adeyemi, Regulation and biotech correspondent
. 1-minute read.
Novartis building on Gran Via Corts Catalanes in Barcelona
A Novartis facility on Gran Via Corts Catalanes in Barcelona, Spain. Photograph: Poniol60/Wikimedia Commons (public domain)

Novartis has disclosed nine reports of tumors in patients treated with Zolgensma (onasemnogene abeparvovec), its gene therapy for spinal muscular atrophy, according to STAT on Thursday. Seven of the nine reports were new disclosures. The cases are being treated as a possible safety signal rather than as proof that the therapy caused the tumors.

In a statement, the company said that since the product's 2019 approval it had logged nine reports of tumors in previously treated patients. Among those, two cases so far showed signs that the tumors could be connected to the gene therapy itself.

Novartis shared the information with neurologists earlier this week, STAT reported. The advocacy group SMA Europe and a Facebook page made the cases public on Thursday. SMA Europe said regulators are now reviewing the reports.

No conclusion has been reached that the tumors were caused by the therapy, and the reports remain under review. The development has worried families, doctors, and patient organizations.

Theo AdeyemiRegulation and biotech correspondent

Theo Adeyemi covers drug regulation and the biotech industry for Clinical Observer. He writes from regulators' notices, company filings and published reporting.

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