Gene therapy at $3.5m a dose approved for US adults with hemophilia B
GuardianSEP 2022
This study's results are promising and could be the first step towards Pfizer's marstacimab revolutionizing treatment for patients with hemophilia A or B. These results suggests the drug could prevent excessive bleeds and reduce treatment burden through once-weekly, under-the-skin administration.
While the experimental therapy is undeniably innovative — as it reduces the amount of anticoagulation instead of addressing the factor deficiency — TFPI blockers in marstacimab can cause clotting episodes that do not usually show up in small studies. The medical community should not get too excited about this development too soon.
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