FDA Investigates Cancer Risks Linked to Novartis Gene Therapy Zolgensma
«Neun Tumormeldungen bei mehr als 6000 Behandelten ergeben keine verlässliche Risikorate,» said Toni Cathomen, director of the Institute for Transfusion Medicine and Gene Therapy at the University Medical Center Freiburg, as reported by tagesspiegel.de.
- The US Food and Drug Administration is reviewing cancer reports in children treated with Zolgensma, a gene therapy for spinal muscular atrophy, according to morgenpost.de.
- Zolgensma, developed by Novartis, delivers a functional SMN1 gene using modified AAV viral vectors and was priced at roughly two million euros, as reported by tagesspiegel.de.
- Two tumor cases are definitively linked to the therapy after vector DNA was found integrated into tumor tissue, per tagesspiegel.de and 20min.ch.
The US Food and Drug Administration is reviewing reports of cancer in children treated with Zolgensma, tagesspiegel.de reported. The therapy, developed by Novartis, is priced around two million and targets spinal muscular atrophy, a rare genetic disease causing muscle wasting that often leads to death before age two, according to morgenpost.de and 20min.ch.
Zolgensma is approved for children under two years old in the US and Europe starting around 2020, as reported by tagesspiegel.de and morgenpost.de. The treatment has also cost roughly two million dollars, according to 20min.ch.
Zolgensma uses a modified virus as a genetic shuttle to deliver a healthy copy of the SMN1 gene into the patient’s cells via a one-time intravenous infusion, morgenpost.de reported. More than 6,000 young patients have received the treatment since 2019, according to tagesspiegel.de.
The FDA noted that tumors occurring after the therapy affected the central and peripheral nervous system, the spinal area, and the kidney, as reported by morgenpost.de. In three of the cases, the oncogene GLI1—which can contribute to the development of cancer—was involved, according to morgenpost.de.
Novartis received nine total reports of tumors among treated patients since 2019, according to tagesspiegel.de and morgenpost.de, with the company releasing a statement on a Friday regarding these reports, per 20min.ch. Toni Cathomen stated that two cases had vector DNA integrated and remain under medical monitoring.
The FDA closely monitors neoplasm risks across AAV-based gene therapy programs while maintaining that the overall benefits of Zolgensma outweigh the potential risks, according to morgenpost.de. Toni Cathomen noted that tumors following AAV gene therapy were observed in mice as early as 2001, tagesspiegel.de reported.
The patient organization SMA Europe urged Novartis to provide clear information on what is known and what this means for treated children, 20min.ch reported. Cathomen emphasized that the gene therapy with Zolgensma saves lives and can considerably improve the children’s mobility, as reported by tagesspiegel.de.
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