- |||||||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Review, Journal: Plasma Clearance of Coagulation Factor VIII and Extension of Its Half-Life for the Therapy of Hemophilia A: A Critical Review of the Current State of Research and Practice. (Pubmed Central) - May 31, 2023 We focus on the EHL FVIII products' structure and function, in particular related to the known discrepancy in results of one-stage clotting (OC) and chromogenic substrate (CS) assays used to assign the products' potency, dosing, and for clinical monitoring in plasma. We suggest a possible root cause of these assays' discrepancy that is also pertinent to EHL factor IX variants used to treat hemophilia B. Finally, we discuss approaches in designing future EHL FVIII variants, including those to be used for hemophilia A gene therapy.
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Review, Journal: Efanesoctocog Alfa: First Approval. (Pubmed Central) - May 22, 2023 Efanesoctocog alfa was approved in February 2023 in the USA for use in adults and children with hemophilia A (congenital FVIII deficiency) for: routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment and control of bleeding episodes; perioperative management of bleeding. This article summarizes the milestones in the development of efanesoctocog alfa leading to this first approval for hemophilia A.
- |||||||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Journal: Altuviiio - a longer-acting factor VIII product for hemophilia A. (Pubmed Central) - May 12, 2023 This article summarizes the milestones in the development of efanesoctocog alfa leading to this first approval for hemophilia A. No abstract available
- |||||||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Now it will be Altuviiio 😊 (Twitter) - Feb 23, 2023
- |||||||||| Altuviiio (efanesoctocog alfa) / SOBI, Sanofi
TREATMENT OF BLEEDING EPISODES WITH EFANESOCTOCOG ALFA IN PATIENTS WITH SEVERE HAEMOPHILIA A IN THE PHASE 3 XTEND-1 STUDY () - Feb 13, 2023 - Abstract #EAHAD2023EAHAD_438; P3 Once-weekly prophylaxis with efanesoctocog alfa showed meaningful improvement in pain and physical functioning in patients with severe haemophilia A. The exit interviews also highlighted additional benefits such as less fatigue and improved health related quality of life. A single 50 IU/kg dose of efanesoctocog alfa effectively treated 96.7% of BEs regardless of bleed type and location in patients receiving prophylaxis or on-demand treatment.
- |||||||||| Altuviiio (efanesoctocog alfa) / SOBI, Sanofi
Journal: Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. (Pubmed Central) - Feb 1, 2023 P3 In patients with severe hemophilia A, once-weekly efanesoctocog alfa provided superior bleeding prevention to prestudy prophylaxis, normal to near-normal factor VIII activity, and improvements in physical health, pain, and joint health. (Funded by Sanofi and Sobi; XTEND-1 ClinicalTrials.gov number, NCT04161495.).
- |||||||||| Altuviiio (efanesoctocog alfa) / SOBI, Sanofi
Efficacy of Efanesoctocog Alfa on Physical Functioning: Results from the XTEND-1 Phase 3 Clinical Trial in Previously Treated Patients with Hemophilia A (ENMCC - Hall D) - Nov 4, 2022 - Abstract #ASH2022ASH_3883; P3 Pain is an important burden in patients with severe hemophilia A. Once-weekly prophylaxis with efanesoctocog alfa resulted in improvement in reduction of pain in patients with severe hemophilia A, when switching from prophylaxis with standard of care FVIII therapies to efanesoctocog alfa. Once-weekly prophylaxis with efanesoctocog alfa resulted in improvement in physical functioning in patients with hemophilia A, positively impacting daily living activities, in patients who were on prior standard of care FVIII prophylaxis.
- |||||||||| efanesoctocog alfa (BIVV001) / SOBI, Sanofi
P1 data, Journal: Efanesoctocog alfa for hemophilia A: results from a phase 1 repeat-dose study. (Pubmed Central) - Apr 27, 2022 Overall, four once-weekly doses of efanesoctocog alfa were well tolerated, no safety concerns identified, and no bleeds reported during the treatment period. Once-weekly efanesoctocog alfa provided high sustained FVIII activity within the normal to near-normal range for 3-4 days post-dose and may improve protection against bleeds in patients with hemophilia A. (EU Clinical Trials Register study 2018-001535-51).
- |||||||||| efanesoctocog alfa (BIVV001) / SOBI, Sanofi
PROPHYLAXIS AND ZERO BLEED FOR ALL ADULT PATIENTS WITH SEVERE HEMOPHILIA: AN ACHIEVABLE AMBITION WITH NEW TREATMENT OPTIONS ([VIRTUAL]) - Jan 28, 2022 - Abstract #EAHAD2022EAHAD_296; After treatment adaptation, 97 out of 99 non-inhibitor patients were on prophylaxis with SHL-FVIII (16), EHL-FVIII (23), EHL-FIX (18), bispecific antibody (n = 40), BIVV001 (n = 2)... In our experience, the wide range of current treatment options as well as the multidisciplinary selection of a personalized treatment modality allowed to achieve the adoption of and adherence to a highly effective prophylaxis in nearly all adult patients with severe HA or HB.
- |||||||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Enrollment closed: XTEND-Kids: Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A (clinicaltrials.gov) - Jan 26, 2022 P3, N=68, Active, not recruiting, In our experience, the wide range of current treatment options as well as the multidisciplinary selection of a personalized treatment modality allowed to achieve the adoption of and adherence to a highly effective prophylaxis in nearly all adult patients with severe HA or HB. Recruiting --> Active, not recruiting
- |||||||||| efanesoctocog alfa (BIVV001) / SOBI, Sanofi
Journal: Molecular determinants of the factor VIII/von Willebrand factor complex revealed by BIVV001 cryo-electron microscopy. (Pubmed Central) - Dec 16, 2021 Our structure shows direct interaction of sulfated Y1680 in FVIII-a3 and VWF-R816, which, when mutated, leads to severe hemophilia A or VWD type 2N, respectively. These results provide insight on this key coagulation complex, explain the structural basis of many hemophilia A and VWD type 2N mutations, and inform studies to further elucidate how VWF dissociates rapidly from FVIII upon activation.
- |||||||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Trial completion: Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A (clinicaltrials.gov) - Dec 6, 2021 P1, N=13, Completed, These results provide insight on this key coagulation complex, explain the structural basis of many hemophilia A and VWD type 2N mutations, and inform studies to further elucidate how VWF dissociates rapidly from FVIII upon activation. Active, not recruiting --> Completed
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