Wednesday, May 29, 2019 Daily Archives

Zolgensma approval marks success for REGENXBIO’s vector platform

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REGENXBIO could receive up to $80 million after the US FDA approved Novartis/AveXis’ gene therapy Zolgensma (onasemnogene abeparvovec), which uses the firm’s NAV Technology Platform. Last week, the US Food and Drug Administration (FDA) approved Zolgensma, a single-dose gene therapy developed by Novartis acquisition AveXis, for the treatment of children less than two years of age with spinal muscular atrophy (SMA). The approval triggered a $3.5 million (€3.1 million) milestone payment to Maryland-based biotech REGENXBIO. Zolgensma is based on REGENXBIO’s…

May 29, 2019

Bavarian Nordic considers turning CMO as it builds out network

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“Having manufacturing capabilities is both a blessing and a noose around your neck,†says Bavarian Nordic as it considers CMO opportunities. Bavarian Nordic has expanded its manufacturing footprint over the past few years, including expanding its bulk vaccine capabilities and adding a fill/finish facility in Kvistgaard, Denmark. The $75 million fill/finish plant is on track to be finalized by the end of the year, with production expected to begin in 2021. The growth of its manufacturing network will support its…

May 29, 2019

Personalized medicines need personalized pricing plans, Orchard

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There is no one-size-fits-all model for cell and gene therapy pricing plans says Orchard Therapeutics, but industry must adapt a system set up for chronic care to incorporate curative one-off treatments. There have been questions over how payors and insurance firms would cope with such next-generation therapeutics have been asked ever since Novartis launched its first cell and gene therapy product Kymriah (tisagenlecleucel) in 2017 at a list price of $475,000. But the recent US Food and Drug Administration (FDA)…

May 29, 2019