5 Key Takeaways

• 1

  The FDA is investigating malignancy risk after molecular testing linked two tumors to integration of Zolgensma vector DNA into patients’ genomes.

• 2

  The FDA received reports of tumors in the central and peripheral nervous systems, spinal and paraspinal regions, and kidney.

• 3

  Three cases involved abnormal GLI1 activity; the mechanism behind GLI1 overexpression in the third case remains under investigation.

• 4

  Novartis reported nine tumor cases among more than 6,000 Zolgensma-treated patients, with vector integration identified in two.

• 5

  The FDA said the risk also applies to Itvisma, reported no tumors after its use to date, and advised monitoring treated patients for tumors.