Health / Other
FDA investigating tumor risk tied to Zolgensma gene therapy
The FDA said it has received postmarketing reports of malignancies in patients treated with the spinal muscular atrophy gene therapy Zolgensma.
Source-checked story: attributed primary-source and published reporting; no on-location reporting.
The Food and Drug Administration said Nov. 6 it has received postmarketing adverse event reports of malignancies in patients treated with Zolgensma, a gene therapy for spinal muscular atrophy, including confirmed reports of product-related insertional oncogenesis involving the GLI1 gene.
The agency said reported tumors include tumors of the central and peripheral nervous systems, spinal/paraspinal regions, and kidney. Three cases involved tumors associated with the GLI1 oncogene, and in two of those cases, molecular analysis showed Zolgensma vector DNA integrated into the host genome, the FDA said.
The FDA said the risk applies to both onasemnogene abeparvovec products, Zolgensma and Itvisma, because they contain the same vector and construct. There have been no reported cases of neoplasia following Itvisma administration to date, the agency said.
The agency said the overall benefits of Zolgensma and Itvisma continue to outweigh their potential risks for their approved uses, though it is evaluating whether regulatory action is appropriate. Novartis, the sponsor, is conducting long-term follow-up safety studies, according to the FDA.
The FDA said patients and clinical trial participants receiving treatment with these products should be monitored for tumor formation.
What we know / what we don't
What we know
- The FDA said it has received postmarketing reports of malignancies in patients treated with the spinal muscular atrophy gene therapy Zolgensma.
What we don't know
- Total number of tumor cases reported.
- Whether labeling changes will follow.
The source record