FDA probes possible tumour link to Novartis gene therapy

what happened

The US Food and Drug Administration said on Friday that it is investigating a risk of neoplasia, including malignancy, linked to Novartis' gene therapy Zolgensma, according to Reuters. Earlier that day, the Swiss drugmaker disclosed two cases in children in which tumours showed evidence of a link to the treatment for spinal muscular atrophy.

Novartis said it had received nine reports of tumours in patients previously treated with Zolgensma since its approval in 2019, and confirmed that more than 6,000 children have received the therapy. The FDA said the overall benefits of Zolgensma and Itvisma still outweigh their risks for approved uses, but it is evaluating whether regulatory action is needed. It reported no cases of neoplasia after Itvisma administration.

why it matters

For investors, the story matters mainly as a regulatory risk for Novartis: an FDA review and possible limits on use could affect expectations for sales of its gene therapy. Tumours alone do not mean a withdrawal, but patient groups' complaints about slow disclosure add uncertainty.

market impact

Акции Novartis: регуляторный риск по генной терапии давит на бумаги компании

• ▼Novartis shares pressure down — an FDA review raises the risk of restrictions on its gene therapy

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