Novartis’s Zolgensma In Spotlight As Second Death Is Probed
Reports of patient deaths could raise questions around safety for Novartis's gene therapy Zolgensma which is expected to get the green light imminently from the FDA.
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Between 600-1,200 people in England and Wales may be able to get Spinraza now that after 17 months of negotiations "appropriate flexibility" has led to an access agreement.
Public statements by Novartis and AveXis suggest Zolgensma could be cost effective at a price up to $5m. Analysts expect $2m. The lower estimate is still stunning, but could it be considered reasonable by comparison?
Novartis may be turning up the rhetoric in spinal muscular atrophy as its cutting-edge gene therapy Zolgensma approaches a market currently enjoyed solely by Biogen’s Spinraza, but Roche is upbeat about the potential of its investigational oral product, risdiplam, to weigh in.