Novartis' OAV101 IT Gene Therapy Shows Motor Function Improvement in Older SMA Patients
• Novartis' gene therapy, OAV101 IT (onasemnogene abeparvovec), has demonstrated improved motor function in older children (2-18 years) with spinal muscular atrophy (SMA) in a late-stage trial.
• The study included SMA Type 2 patients who could sit but never walk independently, showing improvements in sitting, rolling, crawling, and standing compared to a sham control.
• OAV101 IT, which shares Zolgensma's active ingredient, aims to extend Novartis' treatment options for older SMA patients, with plans to present detailed results and discuss with regulators.
• The therapy exhibited a favorable safety profile, with common side effects including upper respiratory tract infection, fever, and vomiting.
Novartis' Intrathecal Zolgensma Shows Positive Phase III Results for SMA Type 2
• Novartis' intrathecal onasemnogene abeparvovec (OAV101 IT) met its primary endpoint in the Phase III STEER study for SMA Type 2.
• The STEER trial demonstrated a statistically significant increase in motor function, as measured by HFMSE scores, in treatment-naïve patients.
• OAV101 IT showed a favorable safety profile, with adverse events similar to the control arm, potentially expanding treatment options for SMA.
• Novartis plans to submit the data to regulatory agencies in 2025, seeking approval to broaden the availability of this one-time gene therapy.
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