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Novartis confirms 9 reported cases of tumors in patients treated with Zolgensma
BioSpace • • business
Key Points:
- Novartis’ gene therapy Zolgensma, used to treat spinal muscular atrophy (SMA), is under scrutiny after reports of nine tumors in treated patients, with two cases showing evidence of vector integration into patient DNA.
- Patient advocates have raised concerns about Novartis allegedly withholding information and conducting a "tiered rollout" of tumor-related findings, prompting calls for more transparent communication.
- Zolgensma’s label already includes warnings about the theoretical risk of tumorigenicity linked to its adeno-associated viral (AAV) vector, but seven tumor cases have not been causally linked to the therapy.
- Over 6,000 patients have received Zolgensma since its 2019 approval, and regulatory authorities, along with patient organizations like SMA Europe, are actively assessing the evidence and urging timely public updates.
- This tumor issue adds to previous safety concerns for Zolgensma, which had been associated with liver-related adverse events, and comes amid broader safety challenges for Novartis, including paused studies of its CAR T therapy rapcabtagene autoleucel due to patient deaths.
