Novartis, CH0012005267

Novartis stock faces FDA review over Zolgensma tumor risk

Published on 11/12/2026 at 11:47 | Editorial responsibility: Rafael Müller, Editor-in-Chief AD HOC NEWS

The FDA is weighing regulatory action over tumor risks linked to Zolgensma. It says the therapy's benefits still outweigh potential risks.

FDA Review Raises Zolgensma Risk

The FDA linked two tumors to integration of Zolgensma vector DNA near or within the GLI1 oncogene.
The review covers Zolgensma and Itvisma because both products use the same vector and genetic construct.
The regulator is assessing labeling and long-term follow-up requirements while maintaining that approved benefits outweigh potential risks.

Novartis, CH0012005267, Illustration mit AI erstellt.

The US Food and Drug Administration is investigating a serious malignancy risk linked to Novartis' Zolgensma gene therapy for spinal muscular atrophy. A safety alert dated Saturday, November 7, 2026, and published by the Hong Kong Department of Health reproduces the FDA's findings. Novartis stock traded at 145.84 euros on Lang & Schwarz at 11:43 CET on Thursday, November 12, 2026.

Gene Therapy Safety Signal

The FDA said postmarketing reports involved tumors in the central and peripheral nervous systems, spinal and paraspinal areas, and the kidney. Three cases involved the GLI1 oncogene, which can promote cancer when overexpressed or abnormally activated.

In two cases, molecular testing found that Zolgensma's adeno-associated virus vector DNA had integrated into the patients' genomes. The therapy's promoter was positioned upstream of or within GLI1. The mechanism behind GLI1 overexpression in a third case remains under investigation.

The agency said the identified risk applies to Itvisma as well as Zolgensma because the products use the same vector and genetic construct. No neoplasia cases had been reported after Itvisma treatment at the time of the alert. The FDA is also monitoring comparable adeno-associated virus programs.

Regulatory Review Continues

The FDA is assessing whether changes are needed to the prescribing information or to long-term follow-up requirements for the two products. Novartis is conducting long-term safety studies. Patients and clinical-trial participants receiving either therapy should be monitored for tumor formation.

The safety issue adds a regulatory overhang to Novartis' gene-therapy portfolio, although the FDA said the overall benefits of Zolgensma and Itvisma continue to outweigh their potential risks for approved uses. The review does not itself announce a label change, withdrawal or restriction.