Novartis hits more problems with Zolgensma; studies halted

The US Food and Drug Administration (FDA) has placed a partial hold on spinal muscular atrophy (SMA) clinical trials for the intrathecal administration of a gene therapy that is already approved as an intravenous treatment.

The intravenous administration of Zolgensma (onasemnogene abeparvovec-xioi) is approved in the USA for the treatment of pediatric patients of less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 gene, despite data manipulation issues emerging after the approval was granted.

Concern at pre-clinical findings