US regulator investigates tumour risk in children given Novartis gene therapy

The US Food and Drug Administration is currently investigating a potential link between neoplasia, or tumour formation including malignancy, and Novartis' gene therapy Zolgensma, according to a recent disclosure. Zolgensma, a treatment for spinal muscular atrophy in children under two years old, has been associated with two previously reported cases of tumours, according to the drug maker.

Novartis has received a total of nine such reports since the therapy's approval in 2019. The FDA has stated that the overall benefits of both Zolgensma and Itvisma, their other gene therapy, continue to outweigh their potential risks for their approved uses, but is evaluating whether regulatory action may be appropriate given the new risk.

There have been no reported cases of neoplasia following administration of Itvisma, which treats patients aged two and older. Zolgensma uses a modified virus to deliver a working copy of a gene into cells, and in the two cases, doctors found evidence that the carrier had inserted its genetic material into the patients' own DNA. Novartis has notified health authorities in a timely manner and included the potential risk of tumours in Zolgensma's prescribing information. The European Medicines Agency has not yet responded to a request for comment.