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- | Hemlibra (emicizumab-kxwh) / Roche, Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Journal: Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumab. (Pubmed Central) - Jan 12, 2025
Specific calibration of the CSA FVIII assay using efanesoctocog and bovine reagents allows for accurate measurement of FVIII activity in patients receiving efanesoctocog, even in the presence of emicizumab.
- || Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
PK/PD data, Review, Journal: Pharmacokinetic evaluation of efanesoctocog alfa: breakthrough factor VIII therapy for hemophilia A. (Pubmed Central) - Jan 3, 2025
Efanesoctocog alfa with outstanding pharmacological properties, well tolerated in the clinical trials, is a promising FVIII therapy for PwHA. Future studies should include long-term safety especially in previously untreated patients.
- ||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
P3 data, Journal, Interview: Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews. (Pubmed Central) - Dec 12, 2024
P3
The exit interviews demonstrated that once-weekly efanesoctocog alfa prophylaxis resulted in patient-relevant and meaningful improvements in pain and physical functioning, consistent with the quantitative findings from XTEND-1. These results support the validity of the Haem-A-QoL PH and PROMIS Pain Intensity 3a assessed during XTEND-1, demonstrating the potential for change with efficacious treatment.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Clinical Outcomes over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study () - Dec 7, 2024 - Abstract #ASH2024ASH_7563;
P3
None of the TEAEs led to death or treatment discontinuation. There were no thrombotic events.ConclusionThe results from over 2 years in previously treated children with severe hemophilia A show that once-weekly efanesoctocog alfa continues to be well tolerated and provides highly effective bleed protection with no FVIII inhibitors reported.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Journal: Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors. (Pubmed Central) - Nov 22, 2024
P3
Efanesoctocog alfa was associated with significantly lower ABRs (any, spontaneous and joint) compared with EHL or SHL prophylaxis therapies. Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Journal: Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia (Pubmed Central) - Nov 22, 2024
P3
Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively. Efanesoctocog alfa prophylaxis was associated with significantly lower rates of any, treated, and joint bleeds and improved joint health compared with emicizumab in patients with severe haemophilia
- |||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
New trial: PROTECT-ALT: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan (clinicaltrials.gov) - Nov 12, 2024
P=N/A, N=120, Not yet recruiting, Sponsor: Sanofi
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Real-World Use of Efanesoctocog Alfa in Adult Patients with Hemophilia a Including Those with a History of Factor VIII Inhibitor (Halls G-H (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_6277;
Our cost analysis is based on estimates, actual costs may differ. Future studies should aim to evaluate long-term comprehensive outcomes in patients on Efa prophylaxis and its impact on health care system utilization.
- Hemlibra (emicizumab-kxwh) / Roche, Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Real-World Patterns of Additional Factor Treatment Use Among Hemophilia a Patients on Regular Prophylaxis in the United States: Results from the Picnichealth Database (Halls G-H (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_4708;
Background The standard-of-care for hemophilia A (HA) involves prophylaxis (PPX) with factor replacement therapies (standard half-life [SHL] and extended half-life [EHL]), or emicizumab (Emi)...Patients with evidence of mild HA (6%?50%), any FVIII inhibitor (on index), or treated with efanesoctocog alfa were excluded...Among Emi PPX users, 62% on weekly regimen, 64% on biweekly regimen, and 36% on monthly regimen used additional factor treatment. Conclusion The majority of patients with moderate or severe HA on routine PPX use additional factor treatments to treat bleeds, whereas a smaller proportion of patients use additional factor treatment before physical activity or procedures to prevent bleeds.
- Nuwiq (simoctocog alfa) / Octapharma
Indirect Comparison of the Efficacy and Therapy-Related Costs of a Pharmacokinetic and Individualized Prophylaxis Regimen with Simoctocog Alfa Versus Other Extended-Half Life Factor VIII Concentrates (Halls G-H (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_4703;
Conclusion The majority of patients with moderate or severe HA on routine PPX use additional factor treatments to treat bleeds, whereas a smaller proportion of patients use additional factor treatment before physical activity or procedures to prevent bleeds. For comparison, aggregated data was obtained from the following trials with EHL concentrates : pathfinder2 (turoctocog alfa pegol, N = 175), A-LONG (efmoroctocog alfa, N = 117), PROTECT FVIII (damoctocog alfa pegol, N = 110), PROPEL (rurioctocog alfa pegol 1
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a (Halls G-H (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_4693;
P2/3, P3
This is attributed to its high-sustained factor activity and reduced factor consumption during the reported perioperative period. The major limitations of the study were : the types of major surgeries varied among studies; the perioperative period data of octocog alfa were not found.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Association between Hemophilia Joint Health Score and Quality of Life Using Results from the Xtend-1 Efanesoctocog Alfa Phase 3 Trial (Room 30 (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_1901;
P3
LS mean difference (95% confidence interval) between QoL PROs CFB at Week 52 in "Maintenance or improvement" and "Worsening" HJHS subgroups were : PROMIS Pain Intensity worst score -0.61 (?1.17 to -0.05; p=0.032), PROMIS Pain Intensity T-score -3.79 (-7.56 to -0.02; p=0.049), Haem-A-QoL total score -6.21 (-12.38 to -0.05; p=0.048), Haem-A-QoL PH score -2.65 (-4.63 to -0.67; p=0.009), and PROMIS PF T-score 3.34 (5.99 to 0.69; p=0.014). Conclusion Our findings indicate a stronger improvement in PROs across the three domains of pain, physical functioning, and overall QoL, in patients with joint improvement or maintenance compared with patients with joint worsening, thus highlighting the importance of improved or preserved joint health in patients with hemophilia A. Prospective validation is warranted to confirm these data.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study (Room 30 (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_1899;
P3
Conclusion Long-term results in adults and adolescents in XTEND-ed shows that once-weekly efanesoctocog alfa continues to provide high efficacy and is well tolerated. No inhibitors were detected and ABRs remained low, with continued improvement observed for overall QoL.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Real-World Experience of Switching to Prophylactic Efanesoctocog Alfa in Patients with Moderate and Severe Hemophilia a: An Analysis of the Adelphi Hemophilia Wave III Disease Specific Programme (Room 29 (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_1894;
The limitations of this study were the small sample size and the limited duration of efanesoctocog alfa treatment. These data indicate that in a real-world setting, efanesoctocog alfa provides an effective alternative to existing treatments for hemophilia A, with a once-weekly dosing schedule.
- || Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
New P1 trial: Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A (clinicaltrials.gov) - Aug 30, 2024
P1, N=24, Not yet recruiting, Sponsor: Swedish Orphan Biovitrum
- |||||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
New trial: ACTIVIIITY: Goal Attainment and Physical Activity in People With Hemophilia A (clinicaltrials.gov) - Jul 30, 2024
P=N/A, N=35, Not yet recruiting, Sponsor: Sanofi
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Journal: Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. (Pubmed Central) - Jul 17, 2024
P3
Efanesoctocog alfa was associated with mainly nonserious adverse events. (Funded by Sanofi and Sobi; XTEND-Kids ClinicalTrials.gov number, NCT04759131.).
- || Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Review, Journal: "Ultra-Long FVIII: A Major Step Forward to a Hemophilia-Free Mind". (Pubmed Central) - Jun 30, 2024
Leveraging expertise gained with fusion to immunoglobulin Fc fragments, disconnecting FVIII from endogenous von Willebrand factor (via a D'-D3 fragment), and benefiting from the pharmacokinetic prolongation provided by the addition of hydrophilic polypeptides, efanesoctocog alfa opens a new era in the treatment of hemophilia A. The term Ultra-Long (UL) FVIII has been proposed to designate it and differentiate it from EHL FVIII. The level of FVIII correction within the normal range for several days provided by this molecule should allow an increasing number of patients to mentally and physically free themselves from the physical and psychological constraints of hemophilia A. Certainly, the constraint of weekly intravenous infusions persists, but is compensated by a correction of hemostasis whose amplitude and duration remain unmatched by other therapeutic options currently available.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Journal: Practical challenges associated with efanesoctocog alfa (ALTUVIIIO) prophylaxis in a 19-month-old male with severe hemophilia A. (Pubmed Central) - Jun 26, 2024
The level of FVIII correction within the normal range for several days provided by this molecule should allow an increasing number of patients to mentally and physically free themselves from the physical and psychological constraints of hemophilia A. Certainly, the constraint of weekly intravenous infusions persists, but is compensated by a correction of hemostasis whose amplitude and duration remain unmatched by other therapeutic options currently available. No abstract available
- |||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Enrollment closed: FREEDOM: A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ?12 Years Treated Once Weekly With Efanesoctocog Alfa (clinicaltrials.gov) - May 22, 2024
P3, N=93, Active, not recruiting, Sponsor: Swedish Orphan Biovitrum
No abstract available Recruiting --> Active, not recruiting
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Pediatric Perspective: A Clinical Discussion on ALTUVIIIO for Kids (Theater 1, Queen Sirikit National Convention Center, Bangkok, Thailand) - May 18, 2024 - Abstract #ISTH2024ISTH_2432;
Recruiting --> Active, not recruiting Sponsored by Sanofi
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Achieving More Days Near Normal with ALTUVIIIO (Theater 1, Queen Sirikit National Convention Center, Bangkok, Thailand) - May 18, 2024 - Abstract #ISTH2024ISTH_2431;
Sponsored by Sanofi Sponsored by Sanofi
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Long-term outcomes with efanesoctocog alfa prophylaxis for previously treated children with severe hemophilia A, an interim analysis of the phase 3 XTEND-ed study (Plenary Hall) - May 17, 2024 - Abstract #ISTH2024ISTH_2095;
P3
The mean (standard deviation [SD]) efficacy period was 35.8 (14.1) weeks. No FVIII inhibitors were detected.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
First interim analysis of clinical outcomes in adults and adolescents with severe hemophilia A receiving efanesoctocog alfa prophylaxis in XTEND-ed, a phase 3 long-term extension study (Plenary Hall) - May 17, 2024 - Abstract #ISTH2024ISTH_2094;
P3
No FVIII inhibitors were detected. A total of 146 patients (including 1 female) rolled over from XTEND-1 to XTEND-ed (12
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi, Nuwiq (simoctocog alfa) / Octapharma
Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States (Exhibition Hall) - May 17, 2024 - Abstract #ISTH2024ISTH_928;
The mean weekly dose was significantly higher in patients treated with simoctocog alfa versus efanesoctocog alfa (98.3 IU/kg vs 52.2 IU/kg; p< 0.001). The median dosing interval in the NuPreviq study was 3.5 days; patients in the XTEND-1 study were treated once-weekly.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Perioperative management with efanesoctocog alfa in adults, adolescents, and children with severe hemophilia A in the phase 3 XTEND clinical program (Ballroom B4) - May 17, 2024 - Abstract #ISTH2024ISTH_668;
P3
All single 50 IU/kg pre-operative doses maintained hemostasis during major surgery. Median (range) number of doses per major surgery was 4.0 (1
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Interim analysis of joint outcomes in adult and adolescent patients with severe hemophilia A receiving efanesoctocog alfa during the phase 3 XTEND-ed long-term extension study (Ballroom B1) - May 17, 2024 - Abstract #ISTH2024ISTH_598;
P3
By Month 12 in XTEND-ed, joint health had improved or been maintained in evaluable participants compared with XTEND-1 baseline, as measured by HJHS total score, total joint score, and subdomain scores (Figure, Table). The HJHS domain with the greatest mean (SD) change from baseline to Month 12 was flexion loss -0.6 (2.7).
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Efanesoctocog alfa treatment outcomes in subjects ?50 years of age from the XTEND-1 trial (Exhibit Hall (Hall 9); Virtual) - Apr 1, 2024 - Abstract #WFH2024WFH_1105;
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Efanesoctocog alfa treatment for children below 12 years of age with severe hemophilia A: Plain language summary of the XTEND-Kids study (Exhibit Hall (Hall 9); Virtual) - Apr 1, 2024 - Abstract #WFH2024WFH_1074;
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Efanesoctocog alfa treatment outcomes in subjects >50 years of age from the XTEND-1 trial (Exhibit Hall (Hall 9); POD B) - Apr 1, 2024 - Abstract #WFH2024WFH_704;
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi, Adynovate (rurioctocog alfa pegol) / Takeda, Advate (octocog alfa) / Takeda
INDIVIDUAL PHARMACOKINETIC EVALUATION OF FIXED-SEQUENCE SINGLE-DOSE OCTOCOG ALFA, RURIOCTOCOG ALFA PEGOL, AND EFANESOCTOCOG ALFA IN ADULTS WITH SEVERE HEMOPHILIA A (Sheraton Ballroom I-II; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_301;
P1
No TEAEs were serious, led to treatment discontinuation, or were deemed related to efanesoctocog alfa treatment.ConclusionConsistent superiority regarding time over 40% FVIII activity, half-life, clearance, and FVIII exposure were observed with efanesoctocog alfa compared with SHL and EHL FVIII in all patients. No serious TEAEs or inhibitor development were reported for efanesoctocog alfa.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi, Nuwiq (simoctocog alfa) / Octapharma
INDIRECT COMPARISON OF PROPHYLAXIS EFFICACY BETWEEN SIMOCTOCOG ALFA AND EFANESOCTOCOG ALFA IN SEVERE HEMOPHILIA A AND THEIR COST IN THE UNITED STATES (Riverwalk AB; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_287;
Based on MAIC, prophylaxis with simoctocog alfa resulted in similar zero bleed rates and total ABR as with efanesoctocog alfa, albeit with a higher weekly dose. Despite higher dosing, simoctocog alfa endured an estimated 29% lower cost burden than efanesoctocog alfa.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
OUTCOMES IN NORTH AMERICAN PARTICIPANTS WHO RECEIVED EFANESOCTOCOG ALFA PROPHYLAXIS IN THE XTEND-1 STUDY (Riverwalk AB; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_243;
P3
Outcomes reported in this subset of participants from North America are similar to those observed in the overall population of XTEND-1. Once-weekly efanesoctocog alfa prophylaxis provided improved bleed protection compared with prior standard-of-care prophylaxis, with improvements in physical health, pain, and joint health.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
DESIGN OF A PROSPECTIVE, OBSERVATIONAL, MULTICENTER STUDY OF THE EFFECTIVENESS OF EFANESOCTOCOG ALFA ON LONG-TERM JOINT HEALTH IN PATIENTS WITH HEMOPHILIA A IN THE UNITED STATES AND JAPAN (Riverwalk AB; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_206;
P
Once-weekly efanesoctocog alfa prophylaxis provided improved bleed protection compared with prior standard-of-care prophylaxis, with improvements in physical health, pain, and joint health. Perioperative use
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
REAL-WORLD EXPERIENCES WITH ALTUVIIIO [ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC- VON WILLEBRAND FACTOR XTEN FUSION PROTEIN]: A FIRST-IN-CLASS TREATMENT OPTION (SPONSORED BY SANOFI) (Chicago Ballroom VI; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_64;
Sponsored by SANOFI Join us for a conversation about the mechanism of extension, pharmacokinetics, efficacy, dosing and safety information from the XTEND-1 clinical trial, as well as real-world clinical cases with ALTUVIIIO. This presentation will feature a live patient perspective.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Journal: Efanesoctocog alfa: the renaissance of Factor VIII replacement therapy. (Pubmed Central) - Feb 15, 2024
The discussion extends beyond regulatory approval to encompass the preclinical and clinical development of efanesoctocog alfa, including considerations for laboratory monitoring. The review also highlights areas that warrant further investigation to deepen our understanding of this groundbreaking therapeutic agent.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Fc-VWF-XTEN Fusion Protein/ALTUVIIIO: 1st year real-world data (N117+N118) - Feb 7, 2024 - Abstract #WFH2024WFH_9;
- || Hemlibra (emicizumab-kxwh) / Roche, Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Review, Journal: Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A. (Pubmed Central) - Jan 12, 2024
While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Phase classification: FREEDOM: A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ?12 Years Treated Once Weekly With Efanesoctocog Alfa (clinicaltrials.gov) - Dec 8, 2023
P3, N=90, Recruiting, Sponsor: Swedish Orphan Biovitrum
Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients. Phase classification: P3b --> P3
- Hemlibra (emicizumab-kxwh) / Roche, Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Cost-Effectiveness of Emicizumab Vs Efanesoctocog Alfa, Standard Half Life (SHL) and Other Extended Half Life (EHL) FVIII Products for Prophylaxis in People with Severe Hemophilia a without ... (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6521;
Advate and Eloctate data were used to represent SHL and EHL, respectively. Findings in this cost-effectiveness analysis suggest that emicizumab is less costly and more effective (i.e., dominant) over a lifetime compared with available FVIII prophylaxis in pediatrics and adult PwHA in the US.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5411;
P3
Once-weekly efanesoctocog alfa provided efficient prophylaxis with no bleeds in 64% of participants, and consistently low ABRs among all types and locations of bleeds. Efanesoctocog alfa provided effective treatment and prevention of bleeds in previously treated children <12 years of age with severe hemophilia A.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated Patients with Hemophilia A from the XTEND-Kids Phase 3 Clinical Trial (SDCC - Room 29) - Nov 3, 2023 - Abstract #ASH2023ASH_3155;
P3
Conclusion Caregivers of children with severe hemophilia A reported major impacts at study baseline on both daily life and functioning with prior treatments. Efanesoctocog alfa prophylaxis provided improvements in both children and their caregiver
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children (SDCC - Room 29) - Nov 3, 2023 - Abstract #ASH2023ASH_3154;
P3
At steady state (Week 26), FVIII activity levels were maintained in the normal to near-normal range (>40 IU/dL) for 3 days and remained >10 IU/dL for ~7 days following a 50 IU/kg dose of efanesoctocog alfa. Once-weekly prophylaxis with efanesoctocog alfa provided high sustained FVIII levels across the week for children <12 years of age in the XTEND-Kids study.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in t... (Marriott Marquis - Pacific Ballroom) - Nov 3, 2023 - Abstract #ASH2023ASH_3116;
Once-weekly prophylaxis with efanesoctocog alfa provided high sustained FVIII levels across the week for children <12 years of age in the XTEND-Kids study. As compared to standard-care factor VIII and at VA-FSS pricing, efanesoctocog alfa prophylaxis is conventionally cost-ineffective for patients with severe hemophilia A. Prophylaxis with efanesoctocog alfa does not meet commonly used distributive equity standards in the United States at this time.
- Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Experience with Accelerometer Physical Activity Tracking in Adults and Adolescents with Hemophilia A: Tool Validation Using Results from the XTEND-1 Efanesoctocog Alfa Phase 3 Trial (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_2808;
P3
Analyses of these results revealed limitations in the psychometric properties of the assessed outcomes. To further understand the personal motivation and barriers for improving physical activity, addition of goal-setting could be considered in future clinical studies.
- | Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Journal: A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories. (Pubmed Central) - Oct 30, 2023
Under- or overestimation occurred with some specific OSAs and most CSAs, which has been previously observed with other modified FVIII replacement products. Efanesoctocog alfa FVIII activity was measured with acceptable accuracy and reliability using several OSA methods and commercial plasma standards.
- ||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Enrollment open: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A (clinicaltrials.gov) - Sep 11, 2023
P=N/A, N=200, Recruiting, Sponsor: Sanofi
Efanesoctocog alfa FVIII activity was measured with acceptable accuracy and reliability using several OSA methods and commercial plasma standards. Not yet recruiting --> Recruiting
- || Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi, Adynovate (rurioctocog alfa pegol) / Takeda, Advate (octocog alfa) / Takeda
PK/PD data, Journal: Pharmacokinetics of recombinant factor VIII in adults with severe hemophilia A: fixed-sequence single-dose study of octocog alfa, rurioctocog alfa pegol, and efanesoctocog alfa. (Pubmed Central) - Aug 4, 2023
P1
Efanesoctocog alfa had 3- to 4-fold longer elimination half-life and 3- to 6-fold greater exposure (area under the FVIII activity-time curve, 6.03 and 3.57 folds) than octocog alfa and rurioctocog alfa pegol. Efanesoctocog alfa provided high sustained FVIII activity in the normal-to-near-normal range (>40 IU/dL) for up to 4 days after the dose and at ?10 IU/dL on day
- |||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi
Clinical: #ISTH2023 Prof @sophie_susen closing the oral communication session with data on patients receiving efanesoctocog alpha! (Twitter) - Jun 28, 2023
- |||| Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / Sanofi
Enrollment open: FREEDOM: A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ?12 Years Treated Once Weekly With Efanesoctocog Alfa (clinicaltrials.gov) - Jun 27, 2023
P3b, N=90, Recruiting, Sponsor: Swedish Orphan Biovitrum
Efanesoctocog alfa provided high sustained FVIII activity in the normal-to-near-normal range (>40 IU/dL) for up to 4 days after the dose and at ?10 IU/dL on day Not yet recruiting --> Recruiting