Novartis reports positive Phase III results for SMA gene therapy OAV101 IT

19 March 2025

Swiss pharma major Novartis (NOVN: VX) has announced positive results from its Phase III STEER study, evaluating the efficacy and safety of intrathecal onasemnogene abeparvovec (OAV101 IT) in patients aged 2 to under 18 with spinal muscular atrophy (SMA) Type 2. 

The study demonstrated a statistically-significant improvement in motor function among participants. Patients receiving OAV101 IT showed a 2.39-point increase on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to a 0.51-point improvement in the sham control group.

The HFMSE is a standard measure for assessing motor abilities and disease progression in SMA patients.

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