The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for VONVENDI®, which expands its indication to include prophylaxis use to reduce the frequency of bleeding episodes in adults with von Willebrand disease (VWD), including those with type 1 and 2 disease, and for on-demand and perioperative management of bleeding in pediatric patients with VWD.

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2025 Bleeding Disorders Conference 

NBDF wrapped up another successful Bleeding Disorders Conference. The policy team was busy with sessions on blood and product safety, access to insurance, and sharing updates on what’s happening in Washington, DC. This year there was also a session dedicated to NBDF’s advocacy efforts focused on women and girls. You can see the highlights from this year’s conference here. 

Pathway to Cures, NBDF’s venture philanthropy fund, is an investor in Be Biopharma, Inc., a clinical-stage company developing engineered B cell medicines.  Be Biopharma recently announced the first participant treated with their therapy BE-101 in the BeCoMe-9 Phase 1/2 clinical trial. 

What is BE-101?

Gene therapy was once hailed as the transformative breakthrough for hemophilia—the long-awaited cure that would change everything. But reality has been more nuanced, and many people in the community have been hesitant to embrace it. Some pharmaceutical companies have canceled their gene therapy programs, removing the treatment from the market. What this means for the future of innovation was what Friday's panel at NBDF's Bleeding Disorders Conference set out to explore.