Bioverativ, a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A.
“People with hemophilia A continue to have a strong unmet need for therapies that can provide protection from bleeds and favorable long-term outcomes in terms of joint health and quality of life with once weekly dosing,” said Tim Harris, Ph.D., D.Sc., executive vice president of research and development at Bioverativ. “In 2014, we made significant progress toward this goal with the introduction of ELOCTATE®, the leading extended half-life therapy for hemophilia A. This IND acceptance expands on that legacy and reflects our continued commitment to advancing scientific innovation that can make a meaningful difference for people with hemophilia.”
BIVV001 builds on the company’s existing 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 investigational factor VIII therapy in development that is designed to overcome the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies.
“BIVV001 is the first molecule of its kind to fuse four different proteins together to address the challenges of hemophilia A,” said Rob Peters, Ph.D., senior vice president of research at Bioverativ. “We are encouraged by our extensive preclinical data which show improved pharmacokinetics that are independent of von Willebrand factor, and look forward to commencing our Phase 1/2a clinical trial later this year.”
Filed Under: Drug Discovery