Biologics

Novartis Reports 9 Tumor Cases in Children Treated With Zolgensma

Novartis has disclosed nine reports of tumors in children treated with its spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), including two cases in which investigators identified evidence of viral vector DNA integration into the patients' genomes, raising new questions about the treatment's long-term cancer risks.

The reports have prompted increased scrutiny of a potential safety signal associated with the adeno-associated virus (AAV) vector used to deliver the therapy. However, a causal relationship between Zolgensma and the reported tumors has not been established.

According to Novartis, more than 6,000 patients worldwide have received Zolgensma through clinical trials, managed access programs and commercial treatment since its 2019 approval.

Of the nine reported tumor cases, two showed evidence of vector DNA integration, a finding relevant to longstanding concerns that insertion of genetic material into a patient's genome could potentially contribute to cancer development. In another case, pathology findings suggested that the tumor was unrelated to treatment. Publicly available information does not establish a causal relationship for the remaining six cases. Novartis said the two patients whose tumors showed vector integration received treatment for their tumors and remain under monitoring.

FDA approved Zolgensma in May 2019 for children younger than two years with SMA and biallelic mutations in the survival motor neuron 1 (SMN1) gene. The one-time intravenous gene therapy uses an AAV9 vector to deliver a functional copy of the SMN1 gene.

The potential for insertional oncogenesis — cancer development resulting from integration of vector DNA into the host genome — has been a recognized theoretical concern for AAV-based gene therapies, although these vectors generally persist predominantly outside chromosomes.

Patient organizations, including SMA Europe and SMA UK, have communicated the findings as regulators evaluate the available evidence.

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Merck's Efinopegdutide Shows MASH Resolution, Fibrosis Improvement

FDA Clarifies Drug Interaction Study Requirements, Labeling Expectations

Rybrevant Faspro OK’d in Aggressive Head and Neck Cancer

Significantly More Unannounced Foreign Inspections