FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B
CBSJUL 2022
The Spin
Narrative A
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.
Narrative B
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.