Evaluating a Novel Human Fibrinogen Concentrate (BT524) for Congenital Fibrinogen Deficiency
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
”Excited to share our new publication evaluating a novel human fibrinogen concentrate (BT524) for the treatment of congenital fibrinogen deficiency!
This fibrinogen concentrate includes three independent virus inactivation and removal steps to ensure maximum viral safety.
In this prospective Phase I/III trial, BT524 was investigated in 27 patients (15 adults and 12 children) with congenital afibrinogenemia or severe hypofibrinogenemia:
- Single-dose administration (70 mg/kg) effectively increased fibrinogen antigen levels from undetectable to therapeutic levels
- Half-life: 67.9 hours (FiAg) and 60.3 hours (FiAc) – providing sustained hemostatic coverage
- Significant improvement in clot firmness (FIBTEM MCF +11.1 mm, p < 0.0001) within 1 hour
- Excellent safety profile – only one mild treatment-related adverse event (increased D-dimer), with no thromboembolic events, hypersensitivity reactions, or inhibitor development.
This new fibrinogen concentrate demonstrates excellent pharmacokinetics while providing an outstanding safety and tolerability profile across all age groups.
An important step forward for patients with this ultra-rare bleeding disorder, and a important improvement in the evolution of fibrinogen replacement therapy.
Congratulations to the study team, the co-authors and Biotest— more exciting work ahead!”
Read the full article here.
Article: Pharmacokinetics, Hemostatic Efficacy, and Safety of a New Human Fibrinogen Concentrate in Adult and Pediatric Patients with Congenital Fibrinogen Deficiency
Authors: Claudia Djambas Khayat, Amal El-Beshlawy, Balkis Meddeb, Abderrahim Khelif, Wolfgang Miesbach, Sonia Adolf, Heike Boehm, Silke Aigner, Salomon Abraha , Fabian Bohlaender, Joerg Schuettrumpf
Stay updated on all scientific advances Hemostasis Today.
-
Jun 25, 2026, 19:32Javier M. Floren: A Blood Clot Is One of the Most Impressive Examples of Teamwork You’ll Ever See
-
Jun 25, 2026, 19:31Marcin Krzysztof Maniak: Linking Tissue Mechanics to Inflammation in Health and Disease
-
Jun 25, 2026, 19:29Deepak Rawat: A Diagnosis Like Immune Thrombocytopenia Changes Your Perspective
-
Jun 25, 2026, 19:27Srikanth R.: Don’t Ignore Your Health While Chasing Success
-
Jun 25, 2026, 19:27Jamie Nash: Delighted to Receive the BBTS Race and Sanger Award 2026
-
Jun 25, 2026, 15:56Wolfgang Miesbach: Building the Future of Gene Therapy – From Centre Readiness to Long-Term Patient Care
-
Jun 25, 2026, 15:46Amber Yates: Making a Meaningful Difference for People Living with Sickle Cell Disease
-
Jun 25, 2026, 15:43Nancy Cavey: Navigating Disability Benefits for Patients with Immune Thrombocytopenia
-
Jun 25, 2026, 15:28Damon Race: Pursuing Long-Term Expression in Hemophilia A Gene Therapy