Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days.
The submission was supported by positive results for BAY94-9027 shown in a Phase 2/3 clinical trial called PROTECT VIII (NCT01580293).
“For more than 25 years, Bayer has been committed to continuously improving disease management for people living with hemophilia A,” David Patti, deputy director, U.S. Pharmaceuticals, and Product Communications at Bayer, said in a statement to Hemophilia News Today. “This filing brings us one step closer to providing another potential therapeutic option for patients who are looking for less frequent dosing.”
Hemophilia A standard-of-care requires regular infusions of factor VIII replacement therapy. But many available therapeutic products have low stability, making factor VIII administration necessary every other day or three times per week.
BAY94-9027 was engineered to overcome this limitation, by adding a small compound called PEG, or polyethylenglycol, to the factor VIII protein. This modification prolongs the stability of the protein circulating in the blood while preserving its coagulation activity.
The PROTECT VIII study, conducted at 97 clinical sites worldwide, evaluated the safety and efficacy of BAY94-9027 in about 140 patients age 12 to 65 with hemophilia A.
“Results from the PROTECT VIII trial showed BAY94-9027 demonstrated protection from bleeds with dosing intervals as few as once every seven days, once every five days, and twice per week,” Patti said.
Data first reported in 2014 showed that BAY94-9027 could achieve bleed protection with extended dosing intervals, and effectively resolved 91% of acute and breakthrough bleed events with one or two infusions.
More recent data from the PROTECT VIII KIDS trial (NCT01775618), presented at the 2016 World Congress of the World Federation of Hemophilia, further demonstrated BAY94-9027’s safety and long-lasting efficacy in preventing and treating bleeding in patients under 12 years of age.
As demonstrated, BAY94-9027 was able to keep bleeds under control in all tested administration regimens — twice weekly, every five days, and every seven days. About 92% of the bleeding events reported during the study were controlled with one or two infusions.
These results were featured in the poster, “PROTECT VIII Kids Trial Results: BAY 94-9027 Safety and Efficacy in Previously Treated Children With Severe Hemophilia A.”