Factor v Deficiency

LACHEN, Switzerland, Jan. 18, 2023 /PRNewswire/ -- Replacement therapy with factor VIII has been the mainstay of care for patients with severe haemophilia A for over 30 years, with proven benefits in terms of bleed prevention and protection of joint health. The availability of emicizumab, a non-factor therapy, in recent years has broadened the scope of management options for these patients, raising questions over how to most effectively and safely use factor VIII together with emicizumab. Treatment decisions need to consider the perspectives of both the patient and the clinician to find the ideal approach for each patient. The interactive Octapharma symposium at the 16th Annual Congress of EAHAD in Manchester, UK, will present the perspectives of three clinicians and three patients on key treatment decisions encountered during the lives of people with haemophilia A. "Understanding the viewpoints of both clinicians and patients is a key component of developing treatment approaches personalised to the lifestyle and clinical condition of each patient" commented Larisa Belyanskaya, Head of IBU Haematology at Octapharma. "We're committed to providing the best treatment options for people with haemophilia A to enable them to live healthy and active lives." The Octapharma symposium "Concomitant use of factor VIII and emicizumab in haemophilia A: Patient cases and clinical trials" will use a highly interactive format to take a close look at three real patient cases. Together with the audience, the international faculty of Dr Guy Young, Dr Carmen Escuriola Ettingshausen and Dr Steve Austin will discuss clinical and patient-related aspects of the diverse cases. The three patients and their families will share their experiences, the challenges they have faced, and decisions that they have taken. In addition to providing their clinical perspectives on the cases, the faculty will present new data and provide updates on ongoing and planned clinical studies of concomitant use of factor VIII and emicizumab in previously untreated patients, patients with factor VIII inhibitors, and patients undergoing major surgery. In addition to this symposium, new data on the binding of Nuwiq® (simoctocog alfa) to platelets in vitro will be presented during the Congress poster sessions on February 8–10, 2023. PO030: In vitro differences in platelet binding among recombinant factor VIII products, presented by Anja Strebel "At Octapharma we're dedicated to providing new health solutions advancing human life," commented Olaf Walter, Board Member. "We appreciate the importance of bringing together the perspectives of both clinicians and people with haemophilia A in order to provide individualised care and improve the lives of patients." About Haemophilia A is an X-linked hereditary bleeding disorder caused by a deficiency of factor VIII (FVIII) which, if left untreated, may lead to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. The disorder affects around one in every 10,000 males worldwide. Prophylaxis with replacement FVIII therapy reduces the number of bleeding episodes and the risk of permanent joint damage. 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 around 10,000 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 180 plasma donation centres across Europe and the US. 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 factor2. Nuwiq® treatment has been assessed in nine2-4 completed clinical trials which included 201 previously treated patients (190 individuals)2 and 108 previously untreated patients3 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1000 IU, 1500 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations5. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups5. 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. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Logo - SOURCE Octapharma AG

LACHEN, Switzerland, Dec. 5, 2022 /PRNewswire/ -- New clinical data from Octapharma's haematology portfolio, including the results of the phase 3 WIL-31 study, will be presented during the Scientific Program at the 64th American Society of Hematology (ASH) Meeting on December 10–13, 2022. Prophylaxis with wilate® significantly reduced bleeding rates in children and adults with any type of von Willebrand disease (VWD) in the phase 3 WIL-31 study. Significantly higher zero bleed rates were observed in haemophilia A patients undergoing pharmacokinetic-personalised prophylaxis with Nuwiq® versus comparator products based on matching-adjusted indirect comparison (MAIC) analyses. The NuPOWER study will investigate the efficacy and safety of Nuwiq® for the management of bleeding during major surgery in patients with haemophilia A on emicizumab prophylaxis. The ATN-06 study will investigate the efficacy, safety and single-dose pharmacokinetics of Atenativ® in patients with congenital antithrombin deficiency undergoing surgery or childbirth. Olaf Walter, Board Member and Head of International Business Units at Octapharma, stated, "We look forward to highlighting our continued efforts in improving the care for people with bleeding disorders." Larisa Belyanskaya, Head of IBU Haematology, added: "The positive results of the WIL-31 study support the use of regular wilate® prophylaxis in people with von Willebrand disease of all ages and all types. The MAIC analysis strengthens the use of personalised prophylaxis with Nuwiq® for bleed management in patients with haemophilia A. The NuPOWER study will give important insight into the use of FVIII in haemophilia A patients on emicizumab during surgery, while the ATN-06 study will provide insights into the prevention of thrombosis in people with congenital antithrombin deficiency during surgery." Aiming for all-round bleed protection in people with haemophilia A and von Willebrand disease and prevention of thrombosis in people with congenital antithrombin deficiency: The phase 3 WIL-31 study investigated the efficacy and safety of wilate® prophylaxis over 12 months in people of all ages with VWD of any type. The primary endpoint was achieved, with an 84% reduction in annualized bleeding rate (ABR) compared with on-demand treatment during the prior run-in study. The median spontaneous ABR decreased by 95%. No serious drug-related adverse events or thrombotic events occurred during prophylaxis with wilate®. The findings indicate that prophylaxis with wilate® is effective in children and adults with VWD of all types. In rare diseases, such as haemophilia A, it is difficult to perform head-to-head studies directly comparing different products. Instead, indirect comparison methods, such as MAIC, can be used to assess the relative treatment effects of different products across studies. A series of MAIC analyses was performed comparing the efficacy of pharmacokinetic-guided, personalised prophylaxis with Nuwiq® with that of personalised prophylaxis regimens with three other FVIII products. The results showed that personalised prophylaxis with Nuwiq® can lead to decreased ABRs and significantly higher rates of patients with zero bleeds compared with comparator products. People with haemophilia A who are receiving emicizumab prophylaxis and need to undergo surgery often require additional haemostatic therapy to control bleeding during and after the invasive procedure. However, there are limited data on the safety and efficacy of using emicizumab and concomitant therapy during surgery. The NuPOWER study will therefore investigate the perioperative efficacy and safety of Nuwiq® in patients receiving emicizumab prophylaxis who undergo major surgery. The study is planned to start in 2023. People with congenital antithrombin deficiency have an increased risk for thrombosis, particularly during high-risk periods such as surgery or childbirth. Anticoagulation therapy (e.g., with an antithrombin concentrate) is therefore advisable in such settings. The ATN-06 study is investigating the use of Atenativ® in people with congenital antithrombin deficiency undergoing surgery or childbirth. Site initiation has begun in 8 countries and 15 sites, and patient enrolment has started. "We are excited to provide updates to the bleeding disorder community at the 64th ASH Meeting," continued Olaf Walter, "The ASH Meeting is an important platform to further demonstrate Octapharma's constant commitment to reducing the burden of living with a bleeding disorder and improving patients' lives". About haemophilia A and VWD Haemophilia A is an X-linked hereditary bleeding disorder caused by a deficiency of factor VIII (FVIII) which, if left untreated, may lead to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. The disorder affects around one in every 10,000 males worldwide. Prophylaxis with replacement FVIII therapy reduces the number of bleeding episodes and the risk of permanent joint damage. VWD is the most common inheritable bleeding disorder caused by deficient or defective clotting protein von Willebrand factor (VWF). Currently, recommended guidelines on the use of prophylaxis are limited. VWD affects males and females equally but is diagnosed more frequently in women due to heavy menstrual bleeding and childbirth. Guidelines for managing these issues are also inadequate. 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 around 10,000 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 180 plasma donation centres across Europe and the US. 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 factor2. Nuwiq® treatment has been assessed in nine2-4 completed clinical trials which included 201 previously treated patients (190 individuals)2 and 108 previously untreated patients3 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1000 IU, 1500 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations5. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups5. About wilate® wilate® is a high-purity human VWF/FVIII concentrate, that undergoes two virus inactivation steps during its production6. No albumin is added as a stabiliser6. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma6. wilate® contains a VWF triplet structure and content of large high molecular weight multimers like normal human plasma6. 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 VWD, when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency)6. 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 - SOURCE Octapharma AG

LACHEN, Switzerland, July 4, 2022 /PRNewswire/ -- Octapharma to present new clinical and scientific data from across Octapharma's haematology and critical care portfolios during the ISTH 2022 Congress Data to be presented in 11 poster presentations and during two Supported Symposia New data from the extensive clinical trial programme with Nuwiq® (simoctocog alfa) to be presented, confirming Nuwiq®'s efficacy and safety profile The latest developments in Octapharma's haematology and critical care portfolios will be presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Congress on July 9–13, 2022, in London, UK. The data will be featured in eleven poster presentations and two Supported Symposia during this major international meeting. Octapharma is proud to be a Gold Supporter of the ISTH 2022 Congress. Poster presentations at the Congress will include data from the Nuwiq® clinical trial programme, including a pooled analysis of long-term data from clinical trials and the first presentation of the results of the global GENA-99 study of Nuwiq in real-world clinical practice. These studies confirm the proven efficacy and safety profile of Nuwiq® in patients with haemophilia A. The safety and immunogenicity results from the phase I/II study of OCTA101, a recombinant FVIII developed for subcutaneous administration, will also be presented, following the recent decision to terminate the study due to safety concerns. Updates from the clinical development programme with the four-factor prothrombin complex concentrate Octaplex® and the high-purity, double virus inactivated, lyophilized human antithrombin III concentrate Atenativ® will also be provided, demonstrating Octapharma's commitment to clinical research and development in critical care. The following posters will be presented. Monday July 11, 18:30–19:30 PB0690. Dose optimisation in children with severe haemophilia A on long-term octanate® prophylaxis. Presenting Author: Evgeny Dmitriev PB0672. Safety and pharmacokinetics of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A. Presenting Author: Sigurd Knaub PB0559. LEX-210: a phase 3, randomized, double-blinded study of four-factor prothrombin complex concentrate in patients with acute major bleeding on direct oral anticoagulant therapy with factor Xa inhibitors. Presenting Author: Ravi Sarode PB0560. Use of heparin-calibrated assays to estimate anti-factor Xa activity of factor Xa inhibitors (FXaI): a literature correlation analysis. Presenting Author: Ravi Sarode PB0716. Prospective, Phase III Study of the Efficacy, Safety, and Pharmacokinetics of a Human Antithrombin III Concentrate in Congenital Antithrombin Deficiency During Surgery or Childbirth. Presenting Author: Cristina Solomon PB0541. Freeze-dried plasma development and assessment of biochemical quality. Presenting Author: Andrea Heger VPB0492. LEX-211 (FARES-II): a phase 3, prospective, active-control randomised study of four-factor prothrombin complex concentrate versus frozen plasma in bleeding adult cardiac surgery patients. Presenting Author: Jeannie Callum Tuesday July 12, 18:30–19:30 PB1149. Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A. Presenting Author: Gita Pezeshki, PB1134. Practical utilisation of Octapharma FVIII concentrates in previously untreated and minimally treated haemophilia A patients entering routine clinical treatment – The Protect-NOW Study. Presenting Author: Susan Halimeh PB1126. Safety and efficacy of simoctocog alfa in patients with haemophilia A in routine clinical practice (GENA-99). Presenting Author: Claire Berger VPB0996. FiiRST-2; a prospective, randomized study of clotting factor concentrates versus standard massive haemorrhage protocol in severely bleeding trauma patients. Presenting Author: Jeannie Callum "We are excited to share our new data and updates at ISTH," said Olaf Walter, Board Member at Octapharma. "Despite advances in the field, managing emergency bleeding and bleeding disorders still comes with considerable challenges. We are constantly striving to address these challenges head-on and to lessen these patients' burdens to improve their day-to-day lives." New data will also be shared during two Supported Symposia as part of Octapharma's 'Under the Spotlight' series. A Key Factor: Aiming for All-Round Bleed Protection in Haemophilia A Saturday July 9, 11:30–12:45 BST Chair: Dr Georgina Hall, John Radcliffe Hospital, Oxford, UK The symposium will focus on new clinical and scientific insights into the use of Nuwiq® to improve the lives of people with haemophilia A. The topics include the use of matching-adjusted indirect comparisons to assess the relative clinical impact of personalised prophylaxis with different FVIII products, and the use of combination therapy to manage bleeding in people with haemophilia A undergoing major surgery. Also presented will be new in vitro data on the binding of FVIII to platelets, and the use of novel RNA sequencing methods to help predict the risk for inhibitor development in children treated for the first time with FVIII. Focus on Females: Patient Experience and Novel Treatment Strategies in Bleeding Disorders Tuesday July 12, 13:15–14:30 BST Chair: Dr Veronica Flood, Medical College of Wisconsin, Milwaukee, USA Women with bleeding disorders face unique challenges; many are left untreated or are diagnosed late, which can increase their bleeding risk and impact quality of life. During the symposium, the distinguished panel will discuss the challenges faced by women and girls with von Willebrand disease and haemophilia A. The speakers will illustrate the need for increased awareness and women-specific clinical data, as well as treatment guidelines to support clinicians in making well-informed treatment decisions for female patients with bleeding disorders. New and ongoing studies with wilate® and Nuwiq® will be presented that aim to improve the management of women with von Willebrand disease or haemophilia A. "We look forward to continuing our efforts in raising awareness around and improving the care for women with bleeding disorders, with the ultimate goal of ensuring that these patients have the ability to receive safe and effective management for their symptoms," commented Larisa Belyanskaya, Head of IBU Haematology 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 around 10,000 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 180 plasma donation centres across Europe and the US. 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 seven completed clinical trials which included 280 patients with severe haemophilia A, of which were 190 previously treated patients (PTPs; 190 individuals) and 90 were previously untreated patients (PUPs) 1. Nuwiq® is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations1. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups1. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production.2 No albumin is added as a stabiliser2. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma2. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma2. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres3. 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)3. Nuwiq® Summary of Product Characteristics. Stadler M et al. Biologicals 2006; 34:281-8. wilate® Summary of Product Characteristics. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Logo - SOURCE Octapharma