FDA lifts year-plus hold on Novartis' Zolgensma, setting the stage for new PhIII
Almost two years after the FDA ordered an abrupt halt to Novartis’ testing of intrathecal delivery of Zolgensma, trials may finally resume.
The Swiss drugmaker says data from its non-human primate toxicology study have resolved all safety concerns regulators had, including any potential risks of dorsal root ganglia, about this route of administration. Novartis’ next step will be initiating STEER, a pivotal Phase III study in SMA type 2 that will enroll “treatment naïve patients who are between two and 18 years of age, able to sit, but have never walked.”
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