Novartis cites previously unknown tumor cases potentially linked to gene therapy

Ed’s stories explore the price, affordability and access of prescription drugs, as well as issues related to patents, litigation and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.

Novartis has acknowledged seven previously unknown cases in which tumors were found in children treated with its spinal muscular atrophy treatment, a surprising development that has raised concerns among families, doctors and patient advocacy groups.

The cases, which for now are seen only as a potential safety signal, were made public Thursday by an advocacy group and on a Facebook page after Novartis disclosed the information to neurologists earlier this week. An advocacy group, SMA Europe, issued a statement saying regulators were currently reviewing the cases.

The drugmaker released its own statement saying that since its treatment – ​​called Zolgensma – was approved in 2019, it has received nine reports of tumors in previously treated patients. Of these, two cases to date have shown that the tumors could be linked to the treatment itself.