Novartis New Data From Single-Dose Gene Therapy Shows Meaningful Efficacy, Safety In Patients With Spinal Muscular Atrophy

On Wednesday, Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA).

In the Phase 3 STEER study, treatment with OAV101 IT led to a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), an assessment of motor ability and disease progression, vs. 0.51 points in the sham control arm.

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Novartis released topline data from the Phase 3 STEER study in December 2024.

In the Phase 3b STRENGTH study, treatment with OAV101 IT in ...