Zolgensma (onasemnogene abeparvovec)
Novartis has presented the Phase 3 trials of Zolgensma (onasemnogene abeparvovec), demonstrating the drug's therapeutic benefit in patients with severe type-1 SMA. Thirty-three patients were included in the study; sitting without support was considered as the primary endpoint. Progression-free survival was considered as the secondary endpoint. Nearly 66% of patients achieved developmental motor milestones after a follow-up of 10.6 months.
19% of patients were able to sit independently for ≥10 seconds; 67% of patients gained head control.
94% of patients were able to swallow liquids, 30% of patients need feeding support, 27% of patients required ventilatory support at baseline.
Evrysdi (risdiplam)
Roche has presented the Phase 2/3 clinical trials of Evrysdi in patients with type 1 spinal muscular atrophy.
Total motor function measure 32 (MFM-32) score at month 12 was considered as the primary endpoint.
59% of patients could sit without support for at least 5 seconds, determined by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development. Among the patients who were alive, 100% of patients were able to swallow, 93% were able to take food orally.
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