Rare Disease Report

FDA Accepts Investigational New Drug Application for Hemophilia A Treatment

JUNE 11, 2017
Mathew Shanley
Earlier today, it was announced that the U.S. Food and Drug Administration (FDA) accepted Bioverativ’s Investigational New Drug (IND) application for BIV001.

BIV001 will allow patients with hemophilia A to be treated once a week instead of the current twice-weekly administration for prophylactic treatment.
The new investigational factor VIII therapy is aimed by global biotechnology company Bioverativ - the hemophilia-focused off-shoot of Biogen - to possibly prolong protection from bleeding with a prophylaxis dosage of once weekly, or longer, for hemophilia A patients. BIVV001 is currently the only molecule designed to fuse 4 different proteins together to meet the challenges of hemophilia A and potentially extend the time between doses.
Hemophilia A, a rare, chronic, genetic disorder, is the result of missing or reduced levels of a protein known as factor VIII, and impairs the ability of patients’ blood to clot. There are an estimated 150,000 patients of the disease worldwide, and because of the disorder, they are susceptible to painful bleeding episodes, irreversible joint damage, and life-threatening hemorrhages.
“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.”
BIV001 was designed with the intent to overcome to 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.”
A phase 1/2 clinical trial is slated to begin in the final quarter of 2017.

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