Novartis Discloses Tumor Reports in Children Treated With Zolgensma
Novartis has disclosed seven previously unknown cases in which tumors were found in children treated with Zolgensma, its gene therapy for spinal muscular atrophy (SMA). The cases have prompted concern among families, doctors and patient advocates, although they are currently regarded only as a potential safety signal rather than proof that the treatment caused the tumors.
Since Zolgensma was approved in 2019, Novartis says it has received nine reports of tumors in patients who previously received the therapy. In two of those cases, the available evidence indicated that the tumors might be linked to the treatment. The company had shared information about the seven newly disclosed cases with neurologists earlier in the week; the cases then became public through an advocacy group and a Facebook page.
SMA Europe said that regulatory authorities are examining the reports. The review is intended to establish whether the tumors represent a pattern associated with Zolgensma or are unrelated events occurring among children treated for a serious inherited disease. The disclosures add to the safety monitoring surrounding a therapy that delivers genetic material to address the underlying cause of SMA.
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