US regulator investigates tumour risk in children given Novartis gene therapy
Quick Look
- The FDA is investigating a risk of neoplasia, including malignancy, associated with Novartis' gene therapy Zolgensma.
- The Swiss drugmaker disclosed two cases of tumors in children linked to the therapy and reported nine tumor cases overall among patients treated since its 2019 approval.
AI-generated summary
Why It Matters
Zolgensma is a Novartis gene therapy used to treat patients less than two years old with spinal muscular atrophy. The therapy was first approved in 2019.
The US Food and Drug Administration said on Friday it was investigating a risk of neoplasia, or tumour formation including malignancy, associated with Novartis' gene therapy Zolgensma.
The Swiss drug maker earlier in the day disclosed two cases in which tumours in children showed evidence of being linked to its gene therapy drug used to treat patients less than two years old with spinal muscular atrophy.
Novartis said that it had received nine reports overall of tumours in patients previously treated with Zolgensma since the therapy was first approved in 2019.
What to Watch
AI outlook — possibilities, not facts
The FDA will issue findings or regulatory action regarding Zolgensma's tumor risk
Likely · Within weeks
Open Questions
- What is the nature and severity of the nine reported tumor cases?
- What specific action will the FDA take as a result of its investigation?
- Are the two disclosed cases fatal or treatable?
