Results were released last night from a longitudinal study of joint health in patients with Hemophilia A treated prophylactically with Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein], marketed as Elocta in Europe and the Middle East.
The interim data were published in the October 30 online edition of Haemophilia
and show participants enrolled in the pivotal Phase 3 ASPIRE study, an extension of A-LONG which was designed to examine the efficacy, safety and pharmacokinetics of Eloctate. Subjects were treated with prophylactic dosing of Elocatate for nearly 3 years and exhibited constant improvement in joint health throughout, regardless of prior treatment regimen, gravity of joint damage or target joints.
Additionally, the post hoc analysis evaluated participants in the Phase 3 A-LONG study designed to examine the efficacy, safety and pharmacokinetics of Eloctate.
Hemophilia A is an uncommon genetic condition in which the blood of a patient is incapable of properly forming clots as the result of missing or reduced levels of Factor VIII. Those who suffer from Hemophilia A experience recurrent painful bleeding episodes, some of which can be life-threatening. Current standard of care includes the previously approved Advate and Kogenate, both for prophylactic use.
Eloctate was approved in the United States in June 2014 for the control and prevention of bleeding episodes, perioperative management and routine prophylaxis in adults and children with hemophilia A.
“Gradual joint destruction, which is the leading cause of morbidity for people with hemophilia, remains a significant challenge in the treatment of hemophilia A,” said Professor Johannes Oldenburg, MD, Institute of Experimental Haematology and Transfusion Medicine at the University of Bonn, Germany, and lead author of the published manuscript in a press release
. “This is the first study to show that functional joint health can continue to improve using prophylactic treatment with an extended half-life factor therapy, even for those who have severe joint disease at the start of treatment.”
The interim analysis evaluated joint health in adult and adolescent participants in both the A-LONG and ASPIRE studies using a modified version of the Hemophilia Joint Health Score (mHJHS), and concluded that joint health improvements were most significant in patients with poor joint health.
In the study, adults and adolescents (n=47) experienced a mean improvement in joint health score of -4.1 at ASPIRE Year 2, compared with A-LONG baseline when treated prophylactically with Eloctate. Notwithstanding pre-study treatment regimen, those enrolled exhibited continuous improvement in mHJHS from A-LONG baseline through ASPIRE Year 2 and benefits were displayed in both subjects with target joints and in subjects with severe joint destruction.
“We are very encouraged by the results of this study as they reinforce the positive impact prophylactic treatment with Elocta has the potential to provide on joint health and patient outcomes,” said Krassimir Mitchev, MD, PhD, Vice President and Medical Therapeutic Area Head of Haemophilia at Sobi. “Along with Bioverativ, we are dedicated to the further study of Fc fusion technology in order to better understand and address the significant unmet needs that remain in hemophilia.”
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