Clinical Hold Lifted on Novartis Gene Therapy

FDA has lifted a partial clinical hold on Novartis’ investigational gene therapy OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA). The decision to lift the hold 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 IT administration, the company says.

Novartis says it now plans to initiate STEER, a global pivotal Phase 3 registration-enabling study of OAV-101 IT in treatment-naïve patients who are between two and 18 years of age, able to sit, but have never walked. “STEER will build upon the Phase 1/2 STRONG study which showed that treatment with OAV-101 IT led to significant increases in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores...,” it says. “Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA.”