In an announcement, Novartis stated that it is working with the US Food and Drug Administration (FDA) to determine the next steps, after data provided by AveXis, the developer of Zolgensma (onasemnogene abeparvovec-xioi), led the regulatory agency to place a ‘partial hold’ on clinical trials for intrathecal administration of the product (injection into the spinal canal or near the brain).
More specifically, the information was related to animal findings in a pre-clinical study that showed dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.
Novartis stated that the decision by the FDA does not impact marketed Zolgensma or ongoing intravenous clinical trials for the gene therapy.
Under the hold, enrollment is paused in the high dose cohort of a multicenter trial testing one-time intrathecal administration of the drug product in patients with spinal muscular atrophy (SMA) Type 2. Low- and mid-dose cohort enrollment for the program is already completed.
Zolgensma was approved by the FDA in May of this year as a therapy for children less than two years old with SMA, with Novartis pricing the drug at $2.1m (€1.88m) – making it the most expensive drug on the market.
One month prior to the approval, Novartis was investigating the death of an infant that participated in the clinical trials.
In August, the FDA announced it had received information from AveXis showing manipulation of data included as part of the company’s biologics license application, with Novartis answering that the evidence it had provided supported an “overall favorable benefit-risk profile” for the product.
A statement from AveXis, published one week later, announced that the company’s founder and CEO, Brian Kaspar, and his brother, Allan, had left the company.