Gene therapy digest - 2021

Gene therapy digest - 2021

2 year hold on Zolgensma’s intrathecal OAV-101 lifted (NOVARTIS)

OAV-101 (SMN1 replacement gene therapy; Phase 3) – Novartis

  • OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in Oct 2019
  • Clinical hold was due to safety concerns from a pre-clinical study which showed dorsal root ganglia (DRG) mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss
  • Decision to lift the hold was based on data from nonclinical toxicology study in non-human primates which addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration
  • Following this decision and input from the FDA and EMA, Novartis now plans to initiate STEER, a global pivotal Phase 3 registration-enabling study
    • Clinical efficacy, safety, and tolerability of OAV-101 IT will be evaluated in treatment naïve patients who are between two and 18 years of age, able to sit, but have never walked (For full story click here)

Novartis 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

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CI Scientists Commentary:

  • OAV-101 is an intrathecal version of Zolgensma, an intravenous approved version for severe patients with “Type 1” SMA
  • Novartis’s Zolgensma has had a rough journey to expanding its access to older patients with SMA with an intrathecal version of the therapy, OAV-101 IT
  • This spinally administered form of the therapy could more than double Zolgensma’s eligible patient pool
  • Novartis’ next step is to consult with the FDA and EMA and initiate STEER

– Dr. Kowndinya, CI Scientists

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