Novartis, CH0012005267

Novartis stock faces FDA review after nine Zolgensma tumor reports

Published on 11/10/2026 at 12:23 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS

Novartis stock faces a safety review after tumor reports tied to Zolgensma. The FDA says benefits still outweigh risks while it considers regulatory action.

FDA review raises safety stakes

The FDA is assessing whether Zolgensma labeling and long-term follow-up studies adequately address the newly identified risk.
The agency says Zolgensma and Itvisma retain a favorable benefit-risk balance for their approved uses.
Novartis remains responsible for monitoring patients and working with regulators on next steps.

Novartis, CH0012005267, Illustration mit AI erstellt.

Novartis stock faces fresh regulatory scrutiny after the U.S. Food and Drug Administration said on November 6 that it was investigating malignancy reports linked to Zolgensma, the company’s gene therapy for spinal muscular atrophy. The FDA said the overall benefits of Zolgensma and the related therapy Itvisma continue to outweigh potential risks for their approved uses.

Novartis said nine patients treated with Zolgensma have developed tumors since the therapy was first approved in 2019, according to BioPharma Dive. Two cases showed evidence that the adeno-associated virus vector used by the treatment had integrated into patients’ DNA. Novartis said both patients received treatment and remain under monitoring.

FDA examines gene therapy risk

The FDA said three tumors were associated with the GLI1 oncogene. In two cases, molecular analysis found Zolgensma vector DNA inserted upstream of or within GLI1, a gene that can contribute to cancer when overexpressed or abnormally activated. The mechanism behind the third case remains under investigation.

The agency said reported tumors involved the central and peripheral nervous systems, spinal and paraspinal regions, and kidney. Its FDA safety communication said the risk also applies to Itvisma because the two products use the same vector and construct. No neoplasia cases had been reported after Itvisma administration at the time of the communication.

The FDA is reviewing whether current prescribing information and long-term follow-up studies are sufficient. The agency advised that patients and clinical trial participants who received either product should be monitored for tumor formation. The potential risk of tumorigenicity from vector integration already appears in the products’ U.S. prescribing information.

What the review means for Novartis

The investigation creates a new safety overhang for Novartis’ gene-therapy franchise, even as the FDA continues to support the treatments’ approved uses. The regulator has not announced a labeling change, restriction or withdrawal and is still evaluating whether action is appropriate.

Novartis stock traded at 145.70 euros on Lang & Schwarz at 12:16 CET on Tuesday, November 10.