Rare Daily Staff

Bioverativ presented initial clinical data from an ongoing phase 1/2 study of its experimental recombinant factor VIII therapy BIVV001 that showed a single low dose extended the half-life of factor VIII to 37 hours with high factor activity levels in patients with hemophilia A.

The data, presented at a late-breaking session at the World Federation of Hemophilia 2018 World Congress being held in Glasgow, Scotland, also showed that BIVV001 was generally well tolerated.

Hemophilia is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired. People with hemophilia A experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Prophylactic injections of factor VIII can temporarily replace the clotting factor that is needed to control bleeding and prevent new bleeding episodes.

BIVV001 is a recombinant factor VIII therapy that is designed to extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. BIVV001 builds on the company’s Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to potentially extend its time in circulation. The company said 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.

BIVV001 was granted orphan drug designation by the Food and Drug Administration in August 2017.

“For decades, scientists have been trying to overcome the von Willebrand factor ceiling, which imposes a limit on the half-life of factor VIII, and these data demonstrate that BIVV001 has finally broken through that ceiling,” said Joachim Fruebis, senior vice president of development at Bioverativ. “Importantly for the hemophilia community, the factor levels seen in this study are unparalleled in hemophilia A.”

May 21, 2018
Photo: Joachim Fruebis, senior vice president of development at Bioverativ