Zolgensma® Safety Update and CPNG Guidance for Families
The U.S. Food and Drug Administration (FDA) recently issued a safety communication regarding reports of tumours in some children treated with Zolgensma® for spinal muscular atrophy. Since the therapy was approved in 2019, Novartis has reported nine tumour cases among more than 6,000 treated children worldwide, with evidence of vector integration identified in two of the three tumours analyzed to date. The Canadian Pediatric Neuromuscular Group (CPNG) recommends continuing routine follow-up and monitoring, as the benefits of treatment continue to outweigh the known risks.
Further information can be found in the following documents:
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