Novartis, CH0012005267

Novartis stock: FDA weighs tumor-risk action for gene therapies

Published on 11/13/2026 at 17:35 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS

Novartis stock remains in focus after an FDA safety review of Zolgensma and Itvisma. The agency says benefits still outweigh potential risks.

FDA review raises gene therapy risks

The FDA linked three tumor cases to the GLI1 oncogene and found vector-DNA integration in two cases.
The agency says Zolgensma and Itvisma benefits still outweigh potential risks for approved uses.
Novartis stock traded at 144.90 euros on Lang & Schwarz on Friday afternoon.

Novartis, CH0012005267, Illustration mit AI erstellt.

On November 6, 2026, the U.S. Food and Drug Administration said it was investigating a serious malignancy risk linked to Novartis' gene therapies Zolgensma and Itvisma. The FDA safety communication said the review follows postmarketing reports of tumors in patients treated with Zolgensma.

What the FDA found

The agency said three cases involved tumors associated with the GLI1 oncogene. In two cases, molecular analysis showed that DNA from the Zolgensma vector had integrated into the patients' genomes, with the construct inserted upstream of or within GLI1. The mechanism behind GLI1 overexpression in the third case remains under investigation.

The reports involved tumors in the central and peripheral nervous systems, spinal and paraspinal regions, and the kidney. The FDA said the risk applies to both onasemnogene abeparvovec products because Zolgensma and Itvisma use the same vector and construct. No neoplasia cases had been reported after Itvisma administration as of the agency's communication.

Regulatory review continues

The FDA said the overall benefits of both therapies continue to outweigh their potential risks for approved uses. It is nevertheless evaluating whether regulatory action is appropriate and reviewing the adequacy of current prescribing information and Novartis' long-term follow-up safety studies.

Both products' U.S. prescribing information describes a theoretical risk of tumorigenicity from integration of AAV vector DNA into the genome. In the meantime, the agency said patients and clinical trial participants receiving the therapies should be monitored for tumor formation. The review creates a regulatory and safety issue for Novartis' gene-therapy portfolio, while the FDA's benefit-risk statement leaves the products' approved uses unchanged for now.

Share price and investor context

Novartis stock traded at 144.90 euros on Lang & Schwarz at 17:32 CET on Friday, November 13. Investors will watch the FDA's assessment of product labeling and long-term monitoring requirements alongside the company's pipeline updates.