top of page

Novartis presented data of Zolgensma; Roche presented data of Evrysdi at the World Muscle Society

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.

Also read: US healthcare system


For pipeline, market overview, and market access reports (syndicate and custo

mized) please write to

For all syndicate reports: Click here



Recent Posts

See All


bottom of page