New Zolgensma data suggest comparable efficacy to Spinraza — analysts
As Novartis awaits the FDA decision on its spinal muscular atrophy (SMA) gene-therapy, Zolgensma, the company that developed the treatment for the rare, deadly inherited disorder, AveXis, presented data snapshots from ongoing trials on Sunday, prompting analysts to suggest that the one-shot therapy was looking comparable to Biogen’s approved Spinraza.
In the ongoing late-stage STR1VE study, designed to evaluate the efficacy and safety of a one-time IV infusion of Zolgensma in patients with SMA Type 1 who are <6 months of age, data showed patients continued to show event-free survival well above normal historical controls, AveXis suggested at the American Academy of Neurology annual meeting.
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