FDA probes tumour risk tied to Novartis gene therapy
NVS•The FDA is investigating tumour risk associated with Novartis’ Zolgensma gene therapy, after the company disclosed two cases with evidence of a link and nine tumour reports overall. Novartis said more than 6,000 children have received the therapy; the FDA said benefits of Zolgensma and Itvisma continue to outweigh potential risks for their approved uses.
1. FDA investigation
The FDA said it was investigating a risk of neoplasia, or tumour formation including malignancy, associated with Novartis’ Zolgensma gene therapy. The agency is evaluating whether regulatory action is appropriate, while saying the benefits of Zolgensma and Itvisma continue to outweigh their potential risks for approved uses.
2. Tumour reports
Novartis disclosed two cases in which tumours in children showed evidence of being linked to Zolgensma and said it had received nine tumour reports overall among patients previously treated with the therapy. In the two cases, doctors found evidence that the treatment’s viral carrier had inserted genetic material into the patients’ DNA, the company said. Novartis said the potential tumour risk is included in Zolgensma’s prescribing information and that it notified health authorities in a timely and appropriate manner.
