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
