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Coagulation Factors

Follow-up to “FDA Approves Hemophilia A Gene Transfer Therapy”

This is a follow-up to “FDA Approves Hemophilia A Gene Transfer Therapy,’ posted July 3, 2023, with reference to Dr. Emmanuel Favaloro’s comment, added August 9 and citing Valentino LA, Kaczmarek R, Pierce GF, et al. Hemophilia gene therapy: first, do no harm. J Thromb Haemost. 2023: S1538-7836(23)00501-9. doi: 10.1016/j.jtha.2023.06.016. [click for a copy]. The […]
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FDA Approves Hemophilia A Gene Transfer Rx

Today, July 3, 2023, the FDA approved the use of BioMarin’s Roctavian® [valoctocogene roxaparvovec] for treating adults with severe hemophilia A without inhibitors. The FDA also approved BioMarin’s companion diagnostic, AAV5 DetectCDx, which identifies the patients most likely to benefit from Roctavian. Click here for the LinkedIn announcement. Click here for a provocative FirstWord PHARMA […]
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Concizumab: Anti-TFPI

On 4-17-2023, Novo Nordisk announced Health Canada’s approval of Alhemo®, their trademark for injectable concizumab, the first anti-tissue factor pathway inhibitor [TFPI] designed to prevent bleeds in Hemophilia A and B patients with inhibitors. The current Canadian approval supports relatively few Hemophilia B [Christmas disease] patients with inhibitors but generalization to Hemophilia A patients could […]
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