Novartis reports Phase III success for SMA gene therapy
Novartis (NOVN: VX) has announced positive Phase III trial results for its investigational gene therapy, onasemnogene abeparvovec, in children and young adults with spinal muscular atrophy (SMA) Type 2.
The STEER study met its primary endpoint, demonstrating improved motor function in patients treated with the therapy, compared to placebo. Adverse events were similar between arms.
Novartis plans to submit the findings to regulators, including the US Food and Drug Administration, in 2025, aiming to bring this one-time gene therapy to market.
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