Novartis announces results from Phase III program for OAV101 IT

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec, OAV101 IT, in a broad population of patients aged two to less than18 years with spinal muscular atrophy, SMA. In the Phase III STEER study, treatment with OAV101 IT led to a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded, HFMSE, a gold standard for SMA-specific assessment of motor ability and disease progression, vs. 0.51 points in the sham control arm. In the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated stabilization of motor function over 52 weeks of follow-up. Novartis plans to file applications with regulatory agencies in H1 2025