Factor VIII (FVIII) replacement therapy as a single agent has been shown in clinical studies and real-world experience to control and prevent bleeding effectively in a variety of settings including surgery1. Efanesoctocog alfa represents a potential new class of FVIII replacement therapy with high sustained factor levels, and the first recombinant FVIII therapy that is designed to be von Willebrand factor (VWF) independent, which extends half-life. It is currently in clinical development and has the potential to offer extended protection with once weekly dosing2, thus addressing an unmet need for people living with severe haemophilia A.
"Efanseoctocog alfa has the potential to advance the treatment of people with haemophilia A" says
Efanesoctocog alfa is currently in phase 3 clinical trials and was granted Fast Track designation by the
XTEND-Kids
XTEND-Kids is an open-label, non-randomised interventional, single-arm study. Participants will receive a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. XTEND-Kids will evaluate efficacy, safety and pharmacokinetics in approximately 65 previously treated patients ≤12 years of age with severe haemophilia A.
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa (rFVIIIFc-VWF-XTEN) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. Efanesoctocog alfa was granted orphan drug designation by the
About the Sobi and
Sobi and
About Sobi™
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across
For more information, please contact
Paula Treutiger, Head of Communication & Investor Relations
+ 46 733 666 599
paula.treutiger@sobi.com
+ 46 767 248 830
maria.kruse@sobi.com
References:
- Srivastava, A., Brewer, A.K., Mauser-Bunschoten, E.P., Key, N.S., Kitchen, S., Llinas, A., Ludlam, C.A., Mahlangu, J.N., Mulder, K., Poon, M.C., Street, A. and (2013), Guidelines for the management of hemophilia. Haemophilia, 19: e1-e47.
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Konkle BA, Shapiro AD,
Quon DV , Staber JM, Kulkarni R, Ragni MV, Chhabra ES, Poloskey S, Rice K, Katragadda S, Fruebis J, Benson CC. BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A. N Engl J Med. 2020 Sep 10;383(11):1018-1027.
XTEN® is a registered trademark of
Postal address SE-112 76
Phone: 46 8 697 20 00 www.sobi.com
https://news.cision.com/swedish-orphan-biovitrum-ab/r/first-patient-dosed-in-phase-3-xtend-kids-study-with-efanesoctocog-alfa-in-children-with-haemophilia,c3324125
https://mb.cision.com/Main/14266/3324125/1400518.pdf
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