Shots:

• The approval is based on ongoing P-III STR1VE & completed P-I START study result assessing Zolgensma (IV) in 21 & 15 patients with SMA and bi-allelic mutation in the SMN1 gene aged <2yrs. respectively
• The collective results: EFS (19 patients survived); 13 reached 14mos. without permanent ventilation- 47.6% & 75.0% achieved the ability to sit without support for≥ 30sec.; 16.7% were able to stand/walk without assistance
• Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy addressing the genetic root cause of SMA- providing a functional copy of SMN gene and has received PRIME designation & accelerated Sakigake designation with its expected approval in EU & Japan in H2’19 respectively

Ref: Novartis | Image: ADP-architects