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Novartis declares Phase III efficacy and safety outcomes of SMA treatment
Novartis has reported promising results from its Phase III clinical program for OAV101 IT (onasemnogene abeparvovec), an investigational gene therapy for spinal muscular atrophy (SMA) in patients aged 2 to under 18.
In the Phase III STEER trial, treatment led to a 2.39-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to 0.51 points in the sham control group, demonstrating significant motor function gains. The therapy also showed positive outcomes in the Phase IIIb STRENGTH trial, which evaluated patients switching from nusinersen or risdiplam, with stabilized motor function over 52 weeks.
With over 170 patients treated and more than 6.4 years of follow-up, Novartis believes this one-time gene replacement therapy could offer lasting benefits by replacing the faulty SMN1 gene. The company remains committed to expanding treatment options for SMA.
22-03-2025