Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

Ad hoc announcement pursuant to Art. 53 LR

  • FDA concluded that OAV-101 intrathecal (IT) clinical program may proceed based on data from nonclinical toxicology study
  • New Phase 3 STEER study will evaluate efficacy, safety, and tolerability of OAV-101 IT in treatment-naïve patients with SMA Type 2 aged between 2 and 18 years old, the first to study gene therapy in this patient population
  • STEER will build upon the OAV-101 IT STRONG study which demonstrated significant increases in HFMSE scores and a …
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