Wolfgang Miesbach: Clinical Evidence for Newly Approved Fibrinogen Concentrate in Congenital Fibrinogen Deficiency
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article he and his colleges co-authored, adding:
“Rare disease, real impact:
Our new paper provides robust clinical evidence for a newly approved fibrinogen concentrate. Congenital fibrinogen deficiency is a rare but serious inherited bleeding disorder. Patients with afibrinogenemia or severe hypofibrinogenemia face life-threatening bleeds — yet robust clinical trial data in this population have long been scarce.
The phase I/III trial provide the comprehensive clinical evidence base for BT524 — now approved as Prufibry in Germany (November 2025) and Fesilty in the USA (December 2025, FDA), with further European approvals expected.
Covering the full age spectrum from in 36 patients 28 days to 75 years, both minor and major bleeds, and both on-demand treatment and peri-operative prophylaxis — this is one of the most comprehensive datasets ever published in this rare disorder:
- 98.9% overall haemostatic success — rated ‘good’ or ‘excellent’ across all age groups and both treatment settings
- Significant and rapid restoration of clot firmness (MCF) after infusion
- Wound healing rated good or excellent in the vast majority of surgical cases
- Excellent safety profile — only 2 mild, non-serious drug-related adverse events; no inhibitory anti-fibrinogen antibodies detected
A heartfelt thank you to all co-authors — Claudia Djambas Khayat, Amal El-Beshlawy, Naglaa Omar, Emna Gouider Belhadjali, Khelif Abderrahim, Sonia Adolf — and to the outstanding Biotest and Grifols team (Silke Aigner, Salomon Abraha, Heike Boehm and Jörg Schüttrumpf).”
Title: Efficacy and safety of prophylaxis and treatment of bleeding events with a novel fibrinogen concentrate from human plasma in patients with congenital fibrinogen deficiency
Authors: Claudia Djambas Khayata, Amal El-Beshlawy, Naglaa Omar, Emna Gouider Belhadj Ali, Abderrahim Khelif, Sonia Adolfe, Wolfgang Miesbach, Silke Aigner, Salomon Abraha, Joerg Schuettrumpf, Heike Boehm
Read the Full Article on Thrombosis Research
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