Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) Receives FDA’s Approval for Spinal Muscular Atrophy in Pediatric Patients

Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) Receives FDA’s Approval for Spinal Muscular Atrophy in Pediatric Patients

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

Click here to read full press release/ article | Ref: Novartis | Image: ADP-architects