Novartis : FDA Lifts Partial Clinical Trial Hold On Spinal Muscular Atrophy Study

 Novartis (NVS) said the U.S. Food and Drug Administration has concluded that OAV-101 intrathecal or IT clinical trials for spinal muscular atrophy or SMA patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.

The FDA's decision was based on data from Novartis' comprehensive nonclinical toxicology study in non-human primates that addressed all issues identified, including questions of dorsal root ganglia injury following intrathecal administration.

Novartis said it 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.

https://www.nasdaq.com/articles/novartis-%3A-fda-lifts-partial-clinical-trial-hold-on-spinal-muscular-atrophy-study-2021-08