Novartis: the FDA is investigating whether Zolgensma has a carcinogenic effect.
Keystone-SDA
The United States Food and Drug Administration (FDA) has launched an investigation to determine if Swiss pharma giant Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec) poses a “serious risk” of malignant tumours developing in patients.
The investigation follows reports issued last week by Novartis regarding tumours detected in patients who had previously used the therapy. Three of the cases involved tumours associated with an oncogene capable of causing cancer if overexpressed.
“In two of these cases, molecular analysis of the tissue demonstrated integration of the Zolgensma adeno-associated viral vector into the host genome,” the FDA stated in a press release.
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The agency considers that the risk of neoplasia associated with Zolgensma – the intravenous version of this treatment for spinal muscular atrophy intended for patients under two years of age – also applies to its intrathecal version for children aged two years and over and adults, marketed under the brand name Itvisma.
Although no adverse events have been reported to date with this second formulation, both versions are based on the same viral vector.
Approved in May 2019 and November 2024 respectively, these two gene therapies still present a favourable benefit-risk profile in the FDA’s view. The agency is considering amending the package leaflet for these products, which currently states “a theoretical risk of tumour formation”.
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Translated from French, reviewed by an English Department journalist.
