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BMN 270 eliminated spontaneous bleeds in patients with hemophilia A
Pasi KJ et al. ASH 2017 Abstract 603
Key clinical point: The investigational gene therapy BMN 270 (valoctocogene roxaparvovec; valrox) eliminated spontaneous bleeds and the need for factor VIII infusions in patients with hemophilia A, and the effects persisted for up to 78 weeks.
Major finding: Median FVIII level was 90 IU/dL at 78 weeks in the higher (6 x 1013 VG/kg) dose cohort.
Data source: A phase I/II, first-in-human study of adenoassociated viral factor VIII gene transfer in 15 patients with severe hemophilia A without inhibitors.
Disclosures: The study was sponsored by BioMarin. Dr. Pasi disclosed research funding, consultancy fees, and speaker and advisory relationships with BioMarin. He disclosed ties to many other companies that develop hemophilia therapies.
Source: Pasi KJ et al. ASH 2017 Abstract 603
Pasi KJ et al. ASH 2017 Abstract 603