FDA Reviews Tumor Reports Linked to Novartis Gene Therapy
The review follows nine reported tumors among Zolgensma patients, including two cases with evidence involving the treatment’s viral carrier.
Key takeaways
- Novartis reported nine tumor cases in patients treated with Zolgensma.
- Two cases showed evidence that viral-carrier DNA had integrated into patients’ genomes.
The FDA is reviewing whether Novartis’ gene therapies Zolgensma and Itvisma carry a tumor risk. The agency’s review followed reports of nine tumors in patients treated with Zolgensma.
Novartis said two cases showed evidence that DNA from the viral carrier used in treatment had integrated into patients’ genetic material. The FDA also cited three tumors involving the cancer-related gene GLI1; the mechanism in one of those cases remains under investigation.
The agency said the tumors have not all been proven to result directly from treatment. It is assessing whether further regulatory action is needed, including changes to safety information or long-term patient monitoring.
Itvisma uses the same viral carrier and genetic structure, but no tumor cases have been reported among its patients, according to the FDA. The agency said the benefits of both treatments outweigh their known risks for approved uses.
This article was produced with the help of AI technology. Based on reporting by the outlets listed above.
