The FINANCIAL — New data demonstrate ELOCTATE [Antihemophilic Factor (Recombinant), Fc Fusion Protein] (marketed as ELOCTA in Europe) and ALPROLIX [Coagulation Factor IX (Recombinant), Fc Fusion Protein] may effectively manage target joint bleeding and maintain low annualized bleeding rates (ABRs) in people with severe hemophilia A and B.
The data, which were presented by Biogen (NASDAQ:BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) at the 57th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla., continue to reinforce the value of extended interval prophylactic dosing of ELOCTATE and ALPROLIX, according to Biogen,
“As the first prolonged half-life therapies, ELOCTATE and ALPROLIX have shown low rates in both joint bleeding and overall annualized bleeding episodes,” said Kate Dawson M.D., vice president, U.S. Medical at Biogen. “Their ability to reduce bleed rates, which may translate into the potential for reducing some joint disease, continues to reaffirm their clinical value for people living with hemophilia A and B.”
For people with severe hemophilia A and B, most bleeding events occur in joints. When bleeding events occur repeatedly in the same joint (known as a target joint), it is often a precursor to chronic joint disease, marked by progressive deterioration of the joint.1 These post-hoc analyses aimed to assess the frequency of bleeding events and the dosing of ELOCTATE and ALPROLIX in study participants who had one or more target joint bleeds [major joint (e.g., knee, elbow, ankle) with three or more bleeding episodes in a three-month (hemophilia B) or six-month (hemophilia A) period].2,3
“Understanding the impact of ELOCTATE and ALPROLIX on people with target joint bleeds provides further insight into their value in a real-world setting,” said Birgitte Volck, M.D., Ph.D., senior vice president of Development and chief medical officer of Sobi. “These new results from the post hoc analyses highlight the value of extended half-life therapy in managing and controlling bleeds, adding to the body of robust clinical data and the longest real-world experience of any extended half-life therapy to date.”
Results Highlight Therapies’ Potential for Reducing Bleed Rates in Target Joints
In this ASPIRE (an ongoing extension of Phase 3 pivotal trials A-LONG and Kids A-LONG) post-hoc analysis, for people with hemophilia A taking ELOCTATE prophylactically, on-study annualized bleeding rates (ABRs) overall and in target joints were lower than pre-study bleeding rates. Data from ASPIRE showed that nearly half of the adult and adolescent participants in the weekly prophylaxis, individualized prophylaxis and modified prophylaxis arms (n=26, n=82, n=12, respectively) did not have any target joint bleeds (42.3, 47.6 and 41.7 percent, respectively). For children, 53.8 percent of participants in the individualized prophylaxis group (n=13) did not have any target joint bleeding episodes. In addition, nearly all (97.4 percent) target joints in adult and adolescent participants taking ELOCTATE were resolved during the follow-up period, suggesting that the therapy may be equally effective for preventing target joint bleeding episodes in weight-bearing and non-weight-bearing joints. The median dosing intervals for ASPIRE participants with target joints at baseline were similar to those for the A-LONG and Kids A-LONG overall population.
In the B-LONG (the pivotal Phase 3 study) post-hoc analysis, for people with hemophilia B taking ALPROLIX prophylactically, the therapy was shown effective in reducing the frequency of bleeding episodes overall and in target joints. The analysis found that 48.6 percent of participants receiving weekly prophylaxis (n=35) and 37.5 percent of participants on individualized interval prophylaxis (n=8) did not have any target joint bleeds at the end of B-LONG. Overall, participants’ target joint, spontaneous target joint and traumatic target joint median ABRs were low for participants in the weekly prophylaxis arm (1.03, 0.00 and 0.00 respectively) and the individualized interval prophylaxis arm (2.20, 2.20 and 0.00 respectively). Additionally, among B-LONG participants entering the trial with target joints (n=43), the on-study median dosing intervals were longer (6.98 days in the weekly prophylaxis arm and 10.25 in the individualized interval prophylaxis arm) than the pre-study dosing interval with a traditional factor therapy, suggesting that target joint bleeds may be effectively managed and controlled with an extended prophylactic dosing regimen.
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