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.
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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