Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. The decision to lift the hold was based on data from Novartis’ comprehensive nonclinical toxicology study in non-human primates (NHP) that addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration. Following this decision and input from the FDA and European Medicines Agency (EMA), Novartis now plans to initiate STEER, a global pivotal Phase 3 registration-enabling study to evaluate the clinical efficacy, safety, and tolerability of OAV-101 IT in treatment naïve patients who are between two and 18 years of age, able to sit, but have never walked. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. The intravenous formulation, Zolgensma® (onasemnogene abeparvovec) is approved in 41 countries. More than 1,400 patients have been treated with Zolgensma IV globally, including in the European Union, South Korea and Canada, where regulatory approval includes dosing guidance for babies and young children up to 21kg.