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

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

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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