Zolgensma: how long were the pivotal tox studies?

“In a 6-month toxicology study conducted in juvenile non-human primates, single IV administration of onasemnogene abeparvovec at the recommended clinical dose level of 1.1 × 10 14 vg/kg, with or without corticosteroid treatment, resulted in microscopic findings in the dorsal root ganglia (DRG), trigeminal ganglia (TG), spinal cord, brainstem, and liver.”

“The Applicant conducted two 12 weeks and one 24 weeks GLP compliant studies with a single IV Zolgensma administration in neonatal FVB wild type mice as indicated in the table below.”

“Route of Administration IV via the temporal vein”