AveXis Receives EC’s Conditional Approval and Activates “Day One” Access Program for Zolgensma (onasemnogene abeparvovec) to Treat Spinal Muscular Atrophy

AveXis Receives EC’s Conditional Approval and Activates “Day One” Access Program for Zolgensma (onasemnogene abeparvovec) to Treat Spinal Muscular Atrophy

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