Baxter International, Inc., announced in December 2012 the Phase I/III study results evaluating the safety and efficacy of BAX 326, a recombinant factor IX (rFIX) product for the treatment and prophylaxis of bleeding episodes for patients with hemophilia B who are more than 12 years old. The data were presented at the 54th Annual Meeting and Exposition of the American Society of Hematology (ASH), held December 8-11, 2012, in Atlanta, GA.
BAX 326 was recently granted orphan-drug designation by the U.S. Food and Drug Administration (FDA). The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drug goes through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available sooner to patients with rare life-threatening diseases who need effective treatments.
The multicenter study investigated the pharmacokinetics, efficacy and safety of BAX 326 in 73 previously treated patients with severe or moderately severe hemophilia B. Results from the study showed that twice weekly prophylactic treatment with BAX 326 achieved a median annualized bleed rate (number of bleeds per year) of 1.99, with 43% of patients experiencing no bleeds. The impact of prophylaxis with BAX 326 also translated into “statistically significant improvements” in physical health-related quality of life (HRQoL).
During the trial, inhibitors were not detected and no cases of anaphylaxis were reported in any patients. Three treatment-related adverse events were reported in 2 of the 73 (2.7%) patients, all of which were mild and transient: dysgeusia (distortion of the sense of taste) occurred twice in one patient and pain in extremity occurred once in another patient. More than 70% of subjects (56 of 73) had 50 or more exposure days to BAX 326 during the study.
“With only one recombinant protein currently available to treat hemophilia B, it was important to focus research efforts on finding another option for patients with this debilitating disease,” said lead investigator Jerzy Windyga, MD, PhD, Institute of Hematology and Blood Transfusion, Warsaw, Poland. “In this study, more than 40 percent of patients using BAX 326 as a prophylactic treatment experienced no bleeds, an important finding given the potentially harmful impact of bleeding episodes for patients.”
Source: Baxter press release dated December 10, 2012