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Sunday, December 2, 2018

ASH: Hemophilia Meds Elocta, Alprolix Efficacious, Safe Over 4 Years


Orphan Biovitrum AB (publ) (Sobi™) and Bioverativ Inc., a Sanofi-company, today announced the final results of ASPIRE and B-YOND, the most comprehensive long-term studies of extended half-life factor therapies in haemophilia. The data from both studies confirm the established safety and sustained efficacy of Elocta® (efmoroctocog alfa), marketed as ELOCTATE® in the United Statesand other countries, and Alprolix® (eftrenonacog alfa) over four years of treatment in previously treated adult, adolescent, and paediatric patients with severe haemophilia A and B, respectively. These results were presented at the 60th Annual Meeting of the American Society of Hematology (ASH).
Factor replacement therapy is the cornerstone of haemophilia care and results from ASPIRE and B-YOND demonstrated that long-term prophylactic treatment with efmoroctocog alfa and eftrenonacog alfa consistently improved annualised bleed rates, including joint bleeds, across all patient populations studied and at extended dosing intervals. No inhibitors were observed in subjects enrolled in either of the two extension studies and the overall safety profile was consistent with the pivotal phase 3 studies. Inhibitor development has been observed with efmoroctocog alfa and eftrenonacog alfa post market.
“These data add to a significant body of evidence showing that efmoroctocog alfa and eftrenonacog alfa provide protection from all types of haemophilia-related bleeds with individualised and flexible dosing regimens across all study populations,” said Tim Harris, Ph.D. DSc., Executive Vice President, Research and Development at Bioverativ. “We remain focused on and committed to providing complete protection for people with haemophilia.”
Debilitating joint disease, which is caused by repeated bleeds into joints over time, is one of the most common complications for people with haemophilia. In ASPIRE and B-YOND, subjects on prophylactic treatment experienced low joint and spontaneous joint annualised bleed rates (ABRs) across all dosing regimens. These results support that prophylactic dosing with efmoroctocog alfa and eftrenonacog alfa can effectively manage and control all types of joint bleeds.
“Joint protection remains a significant challenge in the long-term treatment of haemophilia keeping individuals from living a life without constraints of their disease and these results confirm that our therapies can play a role in the effective prevention of joint bleeds,” said Milan Zdravkovic, Head of Research & Development and Chief Medical Officer at Sobi. “In addition to providing the clinical evidence to support the long-term use of our therapies, we continue to explore the impact of Fc fusion on joint health.”
An interim, post-hoc analysis of ASPIRE published in Haemophilia found that a prophylactic regimen of efmoroctocog alfa can lead to continuous improvement in joint health, regardless of prior treatment regimen, severity of joint damage, or target joints. This retrospective study evaluated joint health in adult and adolescent participants (n=47) using a modified version of the Hemophilia Joint Health Score (mHJHS), a first-line assessment tool that grades joints by specific domains including swelling, muscle atrophy, alignment, range of motion, joint pain, strength and global gait. Additional studies will be needed to confirm these findings and the mHJHS will require further validation.
Efmoroctocog alfa and eftrenonacog alfa are leading extended half-life therapies in the United States and Europe. They have been proven to treat all types of bleeds and can be used in all treatment scenarios, including acute, surgical and emergency situations. The safety and efficacy of both therapies have been studied over hundreds of exposure days in adult, adolescent, and paediatric patients with haemophilia since 2010.

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