{
  "id": 18631970,
  "title": "US regulator investigates tumour risk in children given Novartis gene therapy",
  "url": "https://urgent.news/2026/11/07/us-regulator-investigates-tumour-risk-in-children-given-novartis-gene",
  "topic": "health",
  "section": "Health & Medicine",
  "published": "2026-11-07T03:35:18.000Z",
  "source": {
    "name": "South China Morning Post",
    "slug": "south-china-morning-post",
    "url": "https://www.scmp.com/news/world/united-states-canada/article/3373631/us-regulator-investigates-tumour-risk-children-given-novartis-gene-therapy"
  },
  "original_language": "en",
  "account": "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.",
  "summary": "The US Food and Drug Administration is investigating a risk of neoplasia, or tumor formation including malignancy, associated with Novartis’ gene therapy Zolgensma. The drug is an adeno-associated virus vector-based therapy used to treat pediatric patients less than two years old with spinal muscular atrophy.\n\nNovartis has confirmed nine reports of tumors developing in children treated with the therapy, seven of which were newly disclosed. According to GEN Biotechnology, two of these cases have shown evidence of vector integration into the patients' DNA. Novartis stated that the patients in both cases continue to be monitored and that the company has notified health authorities.\n\nThe potential risk of tumors due to vector integration is currently included in the Zolgensma prescribing information. Novartis reports that the therapy has been used to treat more than 6,000 patients worldwide through commercial treatment, managed access programs, and clinical trials.",
  "key_points": [],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 4,
    "also_reported_by": [
      {
        "outlet": "STAT News",
        "title": "STAT+: A possible comeback for a failed sickle cell gene therapy",
        "url": "https://urgent.news/2026/11/05/stat-a-possible-comeback-for-a-failed-sickle-cell-gene-therapy",
        "published": "2026-11-05T16:22:53.000Z"
      },
      {
        "outlet": "BioPharma Dive",
        "title": "Novartis reveals cancer cases possibly linked to gene therapy Zolgensma",
        "url": "https://urgent.news/2026/11/06/novartis-reveals-cancer-cases-possibly-linked-to-gene-therapy",
        "published": "2026-11-06T16:51:00.000Z"
      },
      {
        "outlet": "GEN Biotechnology",
        "title": "Novartis Confirms Nine Cases of Tumors in Children Treated with Gene Therapy Zolgensma",
        "url": "https://urgent.news/2026/11/06/novartis-confirms-nine-cases-of-tumors-in-children-treated-with-gene",
        "published": "2026-11-06T16:58:32.000Z"
      }
    ]
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}