First Patient Dosed in Avexis' Phase 3 Trial of AVXS-101 in Pre-Symptomatic Spinal...

Discussion in 'Pharma/Biotech Companies - In the News' started by cafead, Apr 25, 2018 at 10:32 PM.

  1. cafead

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    • AVXS-101, has potential to be first-ever one-time gene replacement therapy for spinal muscular atrophy (SMA), a disease which results in early death or lifelong disability with considerable healthcare costs
    • AVXS-101 has Breakthrough therapy designation in the US, PRIME designation in the EU and Sakigake in Japan; expected US patient availability in 2019
    • Novartis and AveXis entered into an Agreement in April, 2018 for Novartis to acquire AveXis for $8.7 billion. Deal expected to close in mid 2018

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