FDA launches investigation of Novartis' Zolgensma after cancer cases

The FDA announced Friday that it has opened an investigation into a “serious risk” of cancer associated with Novartis’ gene therapy Zolgensma, used to treat spinal muscular atrophy. The inquiry follows a disclosure by Novartis the previous day regarding cancer cases observed in patients who received the treatment.

Zolgensma works by delivering a functional copy of a gene to patients, typically infants, with spinal muscular atrophy, a disorder that otherwise leads to progressive muscle weakness. The therapy has been administered since its approval as a one-time treatment intended to address the underlying genetic cause of the disease.

Per Endpoints News, the FDA’s action centers on the agency’s review of this safety signal rather than on any immediate change to the drug’s availability. Novartis’ disclosure of the cancer cases came one day before the FDA’s announcement, according to the report.

No further detail on the number of cases, the mechanism under suspicion, or the timeline for the FDA’s findings was available in the available reporting. The investigation adds to the broader record of safety monitoring for gene therapies, a category of treatment that modifies or supplements a patient’s genetic material and has, in some instances, carried theoretical risks tied to how the introduced genetic material interacts with a patient’s cells.

Endpoints News reported the matter Friday, citing the FDA’s statement and Novartis’ prior disclosure. Further details from regulators or the company were not included in available reporting at time of writing.