Vladimir Ivošev, Senior Business Development Director ar Northway Biotech, shared on LinkedIn:
“BREAKING: 9 Tumors Reported in Patients Treated with Novartis’ Zolgensma
Novartis confirmed to BioSpace that 9 tumors have been reported in patients who were previously treated with Zolgensma, its AAV gene therapy for spinal muscular atrophy (SMA) that was approved in 2019 for children under two. Patient advocates have raised alarms on social media as families learn of the reports directly from their doctors.
What’s Been Confirmed?
- 2 of the 9 cases show evidence of vector integration into the patient’s DNA and both patients have been treated and remain under monitoring.
- Other 7 reports have not been causally linked to the therapy.
- Risk of tumors from vector integration is already included in the prescribing information as a theoretical risk of the AAV vector.
- More than 6,000 patients have been treated across trials, managed access programs, and commercial use.
- SMA Europe says that regulators are actively assessing the evidence and have told Novartis that the SMA community needs timely public communication.
How Did This Come Out?
The news first spread through patient advocates on social media. Parents relayed what their neurologists had told them, including claims that Novartis was rolling out information in tiers. These are claims from families, not findings – but they demonstrate the importance of communication in building trust.
Why This Matters?
- Tumors are a different kind of concern that only long-term follow-up can reveal.
- 2 confirmed cases appear to have occurred 18 to 60 months after dosing, according to one parent.
- Theoretical risk is now showing up in real-world data, even if only in a small number of cases.
In September 2021, an FDA advisory committee examined liver-related adverse events after approximately 1/3 of the ~500 patients treated at that time experienced hepatic events – including one case of serious liver failure. The committee concluded that benefit-risk evaluations of AAV gene therapies should be made on a case-by-case basis.
Big Picture
As gene therapies progress from trials for rare diseases to treating thousands of patients, long-term safety surveillance – especially for latency risks such as insertional mutagenesis – becomes as important as the initial efficacy data. The challenge for the field is to communicate evolving safety information transparently without eroding trust in transformative therapies.
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