BioCentury
ARTICLE | Finance

SMA(ll) wonder

Why AveXis thinks there’s still room for improvement in SMA

December 31, 2016 12:34 AM UTC

It’s a mystery why investors shaved 10% off gene therapy company AveXis Inc. (NASDAQ:AVXS) following FDA’s approval of Spinraza nusinersen for spinal muscular atrophy from Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS). Spinraza has strong efficacy data and a broad label - and will now have a long lead over AveXis’ AVXS-101 - but there’s room for improvement.

FDA approved Spinraza after market close on Dec. 23, quickly turning around an NDA it had accepted in late October. The drug is the first approved for SMA, a rare neurodegenerative disease that is the leading genetic cause of death in infants and toddlers...

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