Hemophilia is a disease in which the blood of those affected cannot coagulate, so any cut or wound can become a major problem and they can suffer from internal and external bleeding. Hence the importance of a new drug to treat haemophilia –efanesoctocog alfa (BIVV001)– whose efficacy and safety data have been presented at the annual Congress of the International Society of Thrombosis and Haemostasis (ISTH) held in London. This drug has shown that it can reduce bleeding by 77% in patients with hemophilia A, compared to usual therapy.
The data revealed in this meeting refer to the results of the phase III trial of BIVV001 for previously treated hemophiliac patients. “This once-weekly drug has a half-life 3-4 times longer than standard and extended half-life factor concentrates. Regarding the pharmacokinetic data, the patients reached a factor level close to normal above 40% during most of the week with an average of 15% at seven days”, explained Dr. Francisco José López Fraile, from the Regional University Hospital of Malaga.
This specialist adds that with the reduction of bleeding “it is achieved that the different sections of quality of life such as pain, physical health and joint health improve significantly”. The data regarding the safety of the drug are also positive because no inhibitor development or severe allergic reactions, anaphylaxis or thrombotic events have been found.
How is the new drug to treat hemophilia
BIVV001 (rFVIIIFc-VWF-XTEN) is a recombinant factor VIII therapy designed to extend protection against bleeding in patients with hemophilia A on a weekly dose. It has been developed by Stockholm-based companies Orphan Biovitrum (STO: SOBI) and French pharmaceutical company Sanofi (Euronext: SAN), and is the first investigational factor VIII therapy shown to overcome the von Willebrand factor ceiling, that limits the half-life in current factor VIII therapies. BIVV001 was designated as an orphan drug by the US Food and Drug Administration (FDA) in August 2017 and by the European Commission in June 2019.
With the reduction of bleeding “it is achieved that the different sections of quality of life such as pain, physical health and joint health improve significantly”
Study results have shown that adults and adolescents treated with efanesoctocog alfa once weekly experienced statistically and clinically significant improvements in physical health, pain intensity, and joint health when compared at week 52 and the start of measurements.
Efanesoctocog alfa was also effective in the treatment of bleeding, including in affected joints; 96.7% of the bleeds resolved with a single dose of 50 IU/kg. The most common side effects (in more than 5% of participants) were headache, arthralgia, falls and back pain.
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