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# Clinical Hold Lifted on Novartis Gene Therapy
- URL: https://www.fdaweb.com/clinical-hold-lifted-on-novartis-gene-therapy/
- Published: 2021-08-03T12:00:00.000Z
- Updated: 2026-09-14T17:08:54.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149848

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.”