Novartis, CH0012005267

Novartis stock faces FDA probe over Zolgensma tumor risk

Published on 11/09/2026 at 12:18 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS

Novartis stock faces new regulatory scrutiny after tumor reports linked to Zolgensma. The FDA says approved benefits still outweigh potential risks.

Safety review broadens investor focus

The FDA is assessing potential tumor risks associated with Zolgensma.
Novartis has reported nine tumor cases among patients treated with the therapy since its 2019 approval.
The agency says the approved benefits of Zolgensma and Itvisma continue to outweigh their potential risks.

Novartis, CH0012005267, Illustration mit AI erstellt.

Novartis faces fresh regulatory scrutiny after the US Food and Drug Administration began investigating a potential tumor risk linked to Zolgensma, the company's gene therapy for spinal muscular atrophy. Reuters reported the development on Monday, November 9, citing the FDA and Novartis.

The FDA said on Friday that it was investigating neoplasia, including malignant tumors, associated with Zolgensma. Novartis had disclosed two cases in which tumors in children showed evidence of a possible link to the therapy. The company said it had received nine tumor reports overall among patients treated with Zolgensma since the product was approved in 2019.

What the safety review covers

Zolgensma is administered by intravenous infusion to children under two years old with spinal muscular atrophy. The therapy uses a modified virus to deliver a working copy of a gene into cells. Novartis said that doctors found evidence in two cases that the viral carrier had inserted genetic material into the patients' DNA.

The treatment has been given to more than 6,000 children, according to the Reuters report. The FDA is evaluating whether regulatory action is appropriate. Itvisma, Novartis' separate gene therapy for patients aged two and older, has not been linked to reported neoplasia, the agency said.

The FDA said the overall benefits of both therapies continue to outweigh their potential risks for their approved uses. Novartis said it had notified health authorities in a timely and appropriate manner and that the potential tumor risk is included in Zolgensma's prescribing information. The company did not provide details on the locations of the two newly disclosed cases.

Investor implications

The review adds a safety and regulatory risk to the outlook for a product serving a rare-disease population. The FDA's benefit-risk assessment limits the immediate interpretation to a review in progress rather than a change to the therapy's approved use. Further regulatory guidance and additional case information are likely to shape the market's assessment of the issue.

Novartis stock traded at 146.83 euros on Lang & Schwarz at 12:12:51 CET on Monday, November 9, 2026. Investors can follow the real-time quote of Novartis stock.