Clinical Trials

Safety concerns halt Novartis gene therapy study

Oct 30, 2019

A study that could lead to an expanded indication for the world’s most expensive drug has been put on hold by the FDA.

Novartis announced this week that the agency halted a study for the use of Zolgensma, a gene therapy that treats spinal muscular atrophy (SMA), after results in an animal trial showed that it could it could trigger dorsal root ganglia mononuclear cell inflammation, which can cause nerve loss or damage.

Zolgensma, which is priced at $2.1 million, has already been approved for patients aged 2 and younger. This new trial was investigating the use of the therapy for children up to the age of 5 who were to take the therapy at a higher dose via spinal infusion.

Novartis said that it is unknown if the results of the animal trial would have an impact on human patients, and said it is working with the FDA to move the study forward.

Read the Reuters report.