Shots:

• The EC’s approval is based on P-III STR1VE-US and P-I START studies assessing the efficacy & safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients aged <6 mos. at dosing- with one/ two copies of the SMN2 backup gene- or two copies of the SMN2 backup gene- respectively
• Zolgensma has demonstrated therapeutic benefit in pre/ symptomatic SMA- including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease and to date- sustained for 5 yrs. post-dosing. Additional supportive data include the results from ongoing P-III SPR1NT study
• AveXis is in talks with EU governments and reimbursement agencies to agree on terms of innovative “Day One” access program- enabling its access in all EU countries. Immediate access to Zolgensma- aligned to the label- is available in France through the ATU framework and expected shortly in Germany

Click here ­to­ read full press release/ article | Ref: Novartis | Image: MDA Strongly