Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular Atrophy

 Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular Atrophy

Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular Atrophy

Shots:

  • The approval is based on P-I START, START long-term follow-up, P-III STR1VE-US, P-III SPR1NT and P-I/II STRONG (intrathecal). START and STR1VE-US assess Zolgensma (one-time, IV infusion) in symptomatic SMA Type 1 patients aged <6mos. with one/two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene respectively
  • The P-III SPR1NT study assess Zolgensma in pre-symptomatic patients aged <6wks. at age of dosing, genetically defined by bi-allelic deletion of SMN1 with 2/3 copies of SMN2. The studies demonstrated clinically benefit in symptomatic and pre-symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in the natural history of the disease 
  • Zolgensma is a one-time gene therapy targeting the genetic root cause of the disease by replacing the function of the missing/ nonworking SMN1 gene with its expected MHLW’s reimbursement by the end H1’20

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

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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