Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis

Start Date28 Nov 2023

Sponsor / Collaborator

Octapharma AG

NCT04030052

/ WithdrawnPhase 3IIT

Emicizumab PUPs and Nuwiq ITI Study

This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI).

Start Date17 Feb 2022

Sponsor / Collaborator

Emory University

[+1]

100 Clinical Results associated with Simoctocog alfa

100 Translational Medicine associated with Simoctocog alfa

100 Patents (Medical) associated with Simoctocog alfa

Literatures (Medical) associated with Simoctocog alfa

01 Sep 2024·THROMBOSIS RESEARCH

Deciphering the circulating microRNA signature of hemophilic arthropathy

Article

Author: Desage, Stephanie ; Marano, Muriel ; Millet, Marjorie ; Leuci, Alexandre ; Chapurlat, Roland ; Dargaud, Yesim ; Lienhart, Anne

BACKGROUND:

Haemophilic arthropathy (HArt) is a serious complication in patients with hemophilia. Early diagnosis and treatment are essential to minimise the development of HArt. The use of biomarkers may improve early diagnosis of HArt. Circulating microRNAs (miRNAs) are small, non-coding RNAsthat regulate gene expression, and are being investigated as promising biomarkers due to their role in joint and bone metabolism.

AIMS:

To investigate differential expression of miRNAs and their relationship to arthropathy in patients with hemophilia A.

METHODS:

miRNA expression was examined in a pilot study followed by a validation study (100 hemophilia A patients with [n = 83] and without HArt [n = 17], 14 controls). Differential miRNA expression was investigated using real-time quantitative PCR.

RESULTS:

The pilot study identified 2 miRNAs differentially expressed in patients with Hart (Pettersson score ≥ 1), after adjusting for the false discovery rate (FDR). The validation study evaluated these 2 miRNAs. The results demonstrated that two miRNAs (miR- 208a-3p and 524-3p) were significantly underexpressed in plasma of patients with HArt compared to patients without arthropathy, with FDR <0.05 (Fig. 1). In addition, 3 miRNAs (130a-3p, miR- and 506-3p) were significantly underexpressed in patients with moderate HArt (Pettersson score 4 to 7).

CONCLUSIONS:

In this proof of concept study we identified a signature of 5 circulating miRNAs associated with Hart with potential as diagnosis tools for HArt. These miRNAs are potential negative regulators of gene expression, suggesting their activity in HArt by interfering with osteoblastic (miR- 208a-3p) and osteoclastic (miR-506-3p) differentiation to impair bone mineralization and remodeling processes, or regulating chondrogenesis (miR-335-5p). miRNAs associated with earlier stages of HArt will be further investigated in a sub-study of the prospective clinical trial PROVE, which will investigate the effects of long-term prophylaxis with simoctocog alfa versus emicizumab in adults with hemophilia A.

01 Mar 2024·European journal of haematology

Clinical efficacy of simoctocog alfa versus extended half‐life recombinant FVIII concentrates in hemophilia A patients undergoing personalized prophylaxis using a matching‐adjusted indirect comparison method

Article

01 Jan 2024·Therapeutic Advances in Hematology

Simoctocog alfa (Nuwiq^®) in children: early steps in life’s journey for people with severe hemophilia A

Review

Author: Klukowska, Anna ; Sidonio, Robert F. ; Knaub, Sigurd ; Young, Guy ; Jansen, Martina ; Mancuso, Maria Elisa ; Bhatnagar, Neha ; Álvarez-Román, María Teresa

People with severe hemophilia A usually experience their first bleed early in life. In children with severe hemophilia A, primary prophylaxis is recommended to prevent recurrent and potentially life-threatening bleeds that significantly impact day-to-day life. Factor VIII (FVIII) prophylaxis is well-established in children and has been shown to reduce the development of hemophilic arthropathy. However, a major challenge of FVIII therapy is the development of neutralizing anti-FVIII antibodies (FVIII inhibitors). Simoctocog alfa (Nuwiq®) is a human cell line-derived recombinant FVIII (rFVIII) whose immunogenicity, efficacy, and safety have been studied in 167 children with severe hemophilia A across two prospective clinical trials and their long-term extensions. In 105 previously untreated children, the inhibitor rate of 16.2% for high-titer inhibitors (26.7% for all inhibitors) was lower than published rates for hamster cell line-derived rFVIII products. There was no inhibitor development in previously untreated children with non-null F8 mutations and in previously treated children. In a case series of 10 inhibitor patients, 8 (80%) underwent successful immune tolerance induction with simoctocog alfa with a median time to undetectable inhibitor of 3.5 months. In an analysis of 96 children who enrolled in the extension studies and received long-term simoctocog alfa prophylaxis for up to 5 years, median spontaneous, joint, and total annualized bleeding rates were 0.3, 0.4, and 1.8, respectively. No thromboembolisms were reported in any of the 167 children, and there were no treatment-related deaths. Optimal care of children should consider several factors, including minimization of inhibitor development risk, maintaining tolerance to FVIII, highly effective bleed prevention and treatment, safety, and impact on long-term outcomes such as bone and joint health. In this context we review the pediatric clinical data and ongoing studies with simoctocog alfa.

News (Medical) associated with Simoctocog alfa

10 Jun 2024

·www.prnewswire.com

Octapharma's continuing dedication to improving the lives of people with rare bleeding disorders to be showcased at ISTH 2024

Octapharma to present new clinical and scientific findings for wilate® and Nuwiq® at the upcoming ISTH 2024 Congress New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia LACHEN, Switzerland, June 10, 2024 /PRNewswire/ -- The most recent developments from Octapharma's Haematology portfolio will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care. Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients' quality of life and mental health. The presentations at ISTH 2024 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs. "Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024." said Olaf Walter, Board Member at Octapharma. "Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients' lives." Oral Presentations Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population. MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study's coordinating investigators, will present an interim analysis of the study. Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial. Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq® can bind to endothelial cell surfaces, improving their functionality. FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A. The oral presentations are scheduled at the following times: Sunday June 23 OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality. Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D Monday June 24 OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability. Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A. Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability. Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C Tuesday June 25 OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3 OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study. Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1 Wednesday June 26 OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4 Poster Presentations Poster presentations are scheduled at the following times: Sunday June 23 PB 0251 - Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States. Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall Tuesday June 25 PB 1102 - Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function. Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall Supported Symposia Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD. Learn About Tomorrow's Advances Today – Breakthrough in All-Round Bleed Protection Sunday June 23, 12:15–13:30 ICT, Room 208 Chair: Guy Young, Children's Hospital Los Angeles and Keck School of Medicine, Los Angeles, California, USA Octapharma's Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A. Next Level Insights on Prophylaxis in von Willebrand Disease – Are You Addressing Your Patient's Needs? Monday June 24, 12:15–13:30 ICT, Room 210 Chair: Craig Kessler, Georgetown University Medical Center, Washington DC, USA This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced. Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress. An opportunity to ask questions will be available at both symposia. Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented: "We are excited to present new insights from the WIL-31 data, and will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A." About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein1. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor1. Nuwiq® treatment has been assessed in nine1 2 3 completed clinical trials which included 201 previously treated patients (190 individuals)1 and 108 previously untreated patients2 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations4. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups4. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser5. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres6. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)6. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US. "Octapharma press releases are specifically for health specialist/medical media and are not for consumer press." [1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. [2]Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. [3] Octapharma AG, data on file. [4] Nuwiq® Summary of Product Characteristics [5] Stadler M et al. Biologicals 2006; 34:281-8. [6] wilate® Summary of Product Characteristics. Logo: SOURCE Octapharma AG

Drug Approval

17 Jan 2024

·www.prnewswire.com

Octapharma to present clinical and scientific data that advances our understanding and treatment of bleeding disorders at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), which takes place in Frankfurt, Germany, from February 6 - 9, 2024

Octapharma will present new clinical and scientific findings for wilate® and Nuwiq® New findings in von Willebrand disease (VWD) and haemophilia A, will be presented by leading experts in a Satellite Symposium, as well as an oral and six poster presentations Presentations will include new data on the efficacy and safety of wilate® prophylaxis in people with von Willebrand disease (VWD) LACHEN, Switzerland, Jan. 17, 2024 /PRNewswire/ -- Showcasing wilate® at EAHAD 2024 WIL-31 is the largest prospective prophylaxis study in VWD with an on-demand run-in study as an intra-individual patient comparator. The study shows that wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD and across examined bleeding sites. The results provide compelling evidence for the use of regular prophylaxis in people with VWD. "The new data from the WIL-31 study provide strong evidence for the use of wilate® prophylaxis in people with all types of VWD. The findings have led to the approval of wilate ® as prophylaxis in the US, thereby expanding the therapeutic options for these patients" – Larisa Belyanskaya, Senior Vice President and Head of IBU Haematology. A deep-dive into the findings from the WIL-31 study will be showcased during Octapharma's Satellite Symposium 'Shifting prophylaxis paradigms in VWD: The WIL-31 study in focus': Jan Astermark (Sweden) will explore the underutilisation of prophylaxis in VWD compared to haemophilia A Robert F. Sidonio Jr. (US) will discuss how the findings from WIL-31 have challenged the status quo of prophylaxis in VWD The efficacy of wilate ® prophylaxis will be explored in three interactive cases of patients in the WIL-31 study: - Child living with VWD presented by Robert F. Sidonio Jr. from Atlanta - Adult living with frequent nosebleeds presented by Ana Boban from Zagreb - Female experiencing heavy menstrual bleeding presented by Csongor Kiss from Debrecen The Satellite Symposium will take place on Wednesday, February 7, 17:30–18:45 (CET), in Panorama Room 2. The importance of preventing frequent nosebleeds in people with VWD, and the efficacy of wilate ® prophylaxis in this setting, will be presented by Ana Boban in the oral SLAM presentation 'Regular prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate is effective for reducing nosebleeds in children and adults with von Willebrand disease' (OR04) on Friday, February 9, 08:30–10:00 CET, Session 6 SLAM. Two poster presentations provide additional insights into the findings of the WIL-31 study: PO213: No accumulation of factor VIII and von Willebrand factor during prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate during the WIL-31 study PO194: Managing von Willebrand disease with inhibitors during prophylaxis with a plasma derived von Willebrand factor/factor VIII concentrate – The WIL-31 study Showcasing Nuwiq® at EAHAD 2024 Beyond its role in the coagulation cascade, emerging evidence indicates that FVIII influences platelet and endothelial cell function. Replacement therapy with recombinant FVIII (rFVIII) concentrates has become a mainstay of treatment for people with haemophilia A; however, it is unknown if there is a difference in how modifications of rFVIII concentrates impact platelet and endothelial cell functionality. Binding of Nuwiq® to activated platelets in vitro was found to be greater compared with other rFVIII concentrates, while in separate research Nuwiq® showed stronger effects on endothelial cell function compared with other rFVIII concentrates: PO027: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet functionality PO024: Investigating the role of factor VIII in endothelial cell function In rare diseases such as haemophilia A, it is not possible to directly compare treatments in a clinical study. In such instances, indirect treatment comparisons can be used to compare the effects of different treatments. One such well-established indirect comparison method is matching-adjusted indirect comparison (MAIC). MAICs were used to compare the efficacy of personalised, pharmacokinetic-guided prophylaxis with Nuwiq® versus other treatments: PO041: Personalised prophylaxis with simoctocog alfa versus standard prophylaxis with efanesoctocog alfa in haemophilia A, a matching-adjusted indirect comparison PO042: Personalised prophylaxis with simoctocog alfa versus standard emicizumab prophylaxis in haemophilia A, a matching-adjusted indirect comparison "Octapharma's continuing commitment to improving the lives of patients is at the core of our values. Supporting clinical and scientific research projects is integral to how we achieve this commitment, and we are excited to present the results of such research at EAHAD 2024" – Olaf Walter, Board Member and Head of International Business Units at Octapharma. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. About Nuwiq ® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein1. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor1. Nuwiq® treatment has been assessed in nine1-3 completed clinical trials which included 201 previously treated patients (190 individuals)1 and 108 previously untreated patients2 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations4. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups4. About wilate ® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production5. No albumin is added as a stabiliser5. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma5. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma5. wilate® is available in 500 IU and 1,000 IU presentations.6 wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)6. The indication for prophylaxis in the US is detailed in the updated prescribing information available here: wilate Full Prescribing Information (wilateusa.com) Octapharma press releases are specifically for health specialist/medical media and are not for consumer press.  References Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. Liesner RJ et al. Thromb Haemost 2021; 121:1400-8. Octapharma AG; Data on file. Nuwiq® Summary of Product Characteristics. Stadler M et al. Biologicals 2006; 34:281-8. wilate® Summary of Product Characteristics. Logo: Contacts: Ivana Spotakova Global Communications Manager ivana.spotakova@octapharma

Drug ApprovalClinical Result

27 Nov 2023

·www.prnewswire.com

Octapharma to present new clinical and scientific data at ASH 2023 from ongoing research initiatives aimed at advancing care for people living with bleeding disorders

LACHEN, Switzerland, Nov. 27, 2023 /PRNewswire/ -- New clinical data from Octapharma's haematology portfolio will be presented during the scientific program at the 65th American Society of Hematology (ASH) Meeting and Exposition, which is being held on December 9–12, 2023, in San Diego, California. New data from the prospective clinical trial using wilate® for prophylaxis in patients with von Willebrand disease (VWD) will be presented. Furthermore, the design of a new phase IV clinical trial of bone and joint health in people with haemophilia A will be presented, in addition to updates from research collaborations between Octapharma and academic institutions. "We are excited to present new data at ASH 2023 highlighting our ongoing commitment to understand the mechanisms underlying bleeding disorders and to improve the standards of care for patients. We are delighted to support clinical and scientific research that advances the care of people with bleeding disorders." – Olaf Walter, Board Member and Head of International Business Units at Octapharma. The WIL-31 study investigated the efficacy and safety of prophylaxis with wilate® in adults and children of any gender with all types of VWD. WIL-31 is the largest prospective prophylaxis study in VWD and the only one with a prospective on-demand run-in study as an intra-individual comparator. The results of the study showed that wilate® prophylaxis is highly effective at reducing the number of bleeds in children and adults and provides compelling evidence to support the use of regular prophylaxis in patients with severe VWD. Data from sub-analyses of the study will be presented at ASH 2023, focusing on the effectiveness of prophylaxis in patients with different types of bleeding and in different subsets of patients. Women with bleeding disorders such as VWD are at an increased risk for heavy menstrual bleeding, but they are underdiagnosed and undertreated. A sub-analysis of WIL-31 will be presented, demonstrating that wilate® prophylaxis was effective in reducing the incidence of heavy menstrual bleeding. An additional sub-analysis focused on the efficacy and safety of wilate® prophylaxis in children and adolescents will also be presented. People with haemophilia A often suffer from joint health issues and reduced bone strength. Prophylaxis with factor VIII replacement therapy has been associated with reductions in joint bleeding rates, joint deterioration, and degenerative joint disease. In contrast, there is a shortage of data on the long-term effects of prophylaxis with the non-factor therapy emicizumab. The new phase IV clinical trial PROVE aims to assess the long-term effects of prophylaxis with Nuwiq® or emicizumab on joint and bone health. The study design will be presented for the first time at ASH 2023. In addition to clinical studies, Octapharma is proud to support scientific investigations with academic research partners that further our understanding of different aspects of bleeding disorders. Results from various avenues of scientific investigation will be presented at ASH 2023. In PUPs with haemophilia A, the development of inhibitors to FVIII is the most significant treatment complication, and there is a need to better understand the mechanisms underlying inhibitor development. The NuProtect study with Nuwiq® was the largest prospective study of a single FVIII product in PUPs. Researchers from University College London, UK, will present new data from a sub-study of the NuProtect study, revealing differences in gene expression profiles between PUPs who developed inhibitors and those who did not. In collaboration with the ETH Zurich and Balgrist Campus Zurich, Switzerland, the effect of FVIII on bone remodelling has been evaluated in vivo, with the results indicating that FVIII and/or von Willebrand can further inhibit the formation of osteoclasts, cells that play a key role in bone remodelling. Platelets and endothelial cells play key roles in haemostasis. Research performed in collaborations with the ETH Zurich, Switzerland, and the University of Eastern Piedmont, Italy, revealed significant differences in the binding of different recombinant FVIII products to platelets and endothelial cells. "We are excited to present additional data from the WIL-31 study, the largest prospective study in VWD, that further supports the use of prophylaxis for children, adolescents and women with von Willebrand disease. We are also proud to introduce the upcoming PROVE study, which will help us understand how best to protect people with haemophilia A from debilitating joint damage." – Larisa Belyanskaya, Head of IBU Haematology. The posters will be presented at the following times: Saturday December 9, 17:30-19:30 (PST) Poster 1255: Design of a Comparative Study (PROVE) To Assess the Long-Term Effects of Prophylaxis With Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia A Patients. Presenting author: Robert Klamroth Poster 1227: Transcriptomic profiling to understand inhibitor development in previously untreated patients with severe hemophilia A. Presenting author: Paul Batty Poster 1244: Impact of recombinant factor VIII and platelet interaction on platelet functionality and hemophilia A treatment. Presenting author: Fabrizio A. Pennacchio Monday December 11, 18:00-20:00 (PST) Poster 3977: Factor VIII is an endothelial factor that promotes vessel stability. Presenting author: Antonia Follenzi Poster 3979: Inter-donor variability in Osteoclastogenesis and responses to Osteoprotegerin (OPG), von Willebrand Factor (vWF), and Factor VIII (FVIII). Presenting author: Joyce Kimenai Poster 3992: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Children and Adolescents with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F. Sidonio, Jr Poster 3997: Efficacy of Regular Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Reducing Heavy Menstrual Bleeding in Females with von Willebrand Disease. Presenting author: Robert F. Sidonio, Jr About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Logo -

Phase 3

100 Deals associated with Simoctocog alfa

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	B02BD02	DB09108

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	European Union	Octapharma AB	22 Jul 2014
Hemophilia A	Iceland	Octapharma AB	22 Jul 2014
Hemophilia A	Liechtenstein	Octapharma AB	22 Jul 2014
Hemophilia A	Norway	Octapharma AB	22 Jul 2014

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
6-Pyruvoyl-Tetrahydropterin Synthase Deficiency	Phase 3	United States	Octapharma AG	01 Jun 2011
6-Pyruvoyl-Tetrahydropterin Synthase Deficiency	Phase 3	Bulgaria	Octapharma AG	01 Jun 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05935358 (NuPOWER, Pubmed \| BMJ Open)	Phase 4	Hemophilia A	28	Simoctocog alfa (Nuwiq) + Simoctocog alfa (Nuwiq) (Severe Haemophilia A)	ilkvmaueqb(guirpyzagj) = lygwlicnit aecinndbca (zberidgdej ) View more	Positive	01 Oct 2025
ISTH2022	Not Applicable	Hemophilia A	-	Simoctocog alfa	cpuxtbpajv(qlegjkxoir) = one was serious (mild pyrexia requiring hospitalisation) but resolved and treatment was continued vuxnipnfmz (jqxzpfxyty )	-	09 Jul 2022
NuProtect (ISTH2021)	Phase 3	Hemophilia A	108	Simoctocog alfa	ztlixjkfky(gfrkgtslnm) = atmqmddvyc bsnxnhches (ddmwezpxmq )	Positive	17 Jul 2021
NCT01992549 (CTgov)	Phase 3	Hemophilia A	48	Human-cl rhFVIII	hbsnadzgcw = wxoodotfcn ipzvrkwrxn (qibyjrzpch, wxhzjkrdjg - znsshvefly)	-	17 Dec 2019
NCT01712438 (CTgov)	Phase 3	Hemophilia A	110	Human-cl rhFVIII (SAF/ITT Population)	gtpfvlfidn = ijppjhxhej haornhagfm (tnuriprnks, nvflfvizly - qyqoqrxafi) View more	-	21 Oct 2019
NCT01712438 (CTgov)	Phase 3	Hemophilia A	110	Human-cl rhFVIII (PROPH Population)	pqfolfawpf(umsgkudjca) = gfljfdjujp anmlpelkyk (kvaxiccpka, jnmtoagcdk - yseqzmzydh)	-	21 Oct 2019
AHEAD International and German Studies (ISTH2019)	Not Applicable	Hemophilia A	1,099	rAHF ITI therapy	wxvahgvkoh(rjgypzmift) = schxnlbqgd bzioyfazvl (lgvnqxeqym )	Positive	10 Jun 2019
NCT01863758 (CTgov)	Phase 3	Hemophilia A	66	Human-cl rhFVIII	iyunthaoxs(jqqzbjytwm) = crgeywnpme xaiehubyff (lvzrcxigrl, 13.43) View more	-	30 Jan 2018
NCT01341912 (CTgov)	Phase 3	Hemophilia A \| 6-Pyruvoyl-Tetrahydropterin Synthase Deficiency	3	Human-cl rhFVIII	adelrvksjx = pdusoatuey kvighjessp (xnajdevcjt, zinlnmgyry - ilcatvtfnm) View more	-	03 Feb 2014
NCT00989196 (CTgov)	Phase 2	Hemophilia A	22	Kogenate FS	nvbspnukne(kkkilxnlmn) = fxvveslaxj mxyhtfkrsn (pwaixvrrdo, 0.09) View more	-	17 Jan 2014

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