Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin Free Method] for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A. ADVATE is the only antihemophilic factor approved in the United States for prophylactic use in both adults and children.
The approval is based on a Phase IV prophylaxis study sponsored by Baxter demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates (ABR) in hemophilia A patients from 44 to one as compared to an on-demand regimen. Forty-two percent of study patients experienced zero bleeds during one year on prophylaxis. Of the two prophylactic regimens approved for use, the dosing schedule of every three days, a pharmacokinetic-driven regimen based on patient's clinical response, offered some patients the option of fewer infusions over one year of treatment.
"Emerging data provide important information to help physicians optimize care for hemophilia patients of all ages by preventing unexpected bleeding events that can have a detrimental impact on the lives of patients," said Leonard Valentino, M.D., Director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. "These data confirm the important clinical benefits of ADVATE when used as a prophylactic therapy to reduce bleeding episodes."
The study findings demonstrated a statistically significant reduction in the median annual bleeding rate, with patients experiencing 44 bleeds (per patient per year) during on-demand treatment compared to one bleed (per patient per year) while on either of the prophylactic regimens evaluated, a 98 percent reduction in annual bleed rate (p<0.0001). Nearly half (42 percent) of patients experienced no bleeding episodes while on one year of prophylactic treatment. Evaluable patients had greater than or equal to 90 percent adherence to the prescribed prophylactic regimen. While the trial was not powered to demonstrate equivalence in bleeding rate between the two prophylaxis arms, there was no statistically significant difference in bleeding frequency observed between the two prophylaxis regimens studied.
"This latest clinical milestone for ADVATE is an important step forward for people living with hemophilia A as we continue to research ways to advance care for this patient population," said Bruce Ewenstein, M.D., Ph.D, vice president, clinical affairs, Baxter's BioScience business. "This rigorous clinical study demonstrated that the number of bleeding episodes experienced each year could be reduced to as low as one event with prophylactic treatment. Further, the pharmacokinetic-driven dosing regimen based on patient's clinical response every third day, offers some patients the option of fewer infusions over one year of treatment than the current standard prophylaxis regimen."
Baxter International Inc.