Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review
Abstract Damoctocog alfa pegol (BAY 94-9027, Jivi®), is a site-specifically PEGylated, extended half-life recombinant factor VIII (FVIII) that is approved in several European and non-European countries for on-demand treatment and prophylaxis of bleeding in previously treated patients aged ≥ 12 year...
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| Format: | Article |
| Language: | English |
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Adis, Springer Healthcare
2024-08-01
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| Series: | Drugs in R&D |
| Online Access: | https://doi.org/10.1007/s40268-024-00481-7 |
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| author | Mark T. Reding Shadan Lalezari Gili Kenet Giovanni Di Minno Jonathan Ducore Alexander Solms Anita Shah Pål André Holme Lone H. Poulsen Karina Meijer Mindy Simpson Maria Elisa Mancuso |
| author_facet | Mark T. Reding Shadan Lalezari Gili Kenet Giovanni Di Minno Jonathan Ducore Alexander Solms Anita Shah Pål André Holme Lone H. Poulsen Karina Meijer Mindy Simpson Maria Elisa Mancuso |
| author_sort | Mark T. Reding |
| collection | DOAJ |
| description | Abstract Damoctocog alfa pegol (BAY 94-9027, Jivi®), is a site-specifically PEGylated, extended half-life recombinant factor VIII (FVIII) that is approved in several European and non-European countries for on-demand treatment and prophylaxis of bleeding in previously treated patients aged ≥ 12 years with hemophilia A. Reliable measurements can be obtained using most one-stage and chromogenic FVIII assays over a wide concentration range. The efficacy, safety and pharmacokinetics (PK) of damoctocog alfa pegol have been studied extensively in the PROTECT VIII clinical trials, and its long-term safety and effectiveness profile is continuing to build through observational and interventional real-world studies. The PK of damoctocog alfa pegol was shown to be improved as compared with that of sucrose-formulated rFVIII (rFVIII-FS, Kogenate®), and was also demonstrated to be non-inferior to and, for some variables, more favorable than rFVIII-Fc fusion protein, efmoroctocog alfa (Elocta®; NCT03364998), rurioctocog alfa pegol (BAX 855, Adynovate®/Adynovi®; NCT04015492), and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, Advate®; NCT02483208). Damoctocog alfa pegol was generally well tolerated and none of the patients in any of the clinical trials, including the PROTECT VIII clinical program, HEM-POWR, or ongoing single-center studies, developed FVIII inhibitors. Efficacy for perioperative hemostasis has been demonstrated. Low bleeding rates were achieved across the studies, with twice weekly, every 5-day and every 7-day prophylaxis offering patients ≥ 12 years and their clinicians the chance to tailor treatment to individual needs and lifestyles, while maintaining long-term protection from bleeds and their consequences. Graphical Abstract |
| format | Article |
| id | doaj-art-5d2cb8c1b4234747b684fe4c71fb9d09 |
| institution | Kabale University |
| issn | 1174-5886 1179-6901 |
| language | English |
| publishDate | 2024-08-01 |
| publisher | Adis, Springer Healthcare |
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| series | Drugs in R&D |
| spelling | doaj-art-5d2cb8c1b4234747b684fe4c71fb9d092025-01-05T12:33:24ZengAdis, Springer HealthcareDrugs in R&D1174-58861179-69012024-08-0124335938110.1007/s40268-024-00481-7Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product ReviewMark T. Reding0Shadan Lalezari1Gili Kenet2Giovanni Di Minno3Jonathan Ducore4Alexander Solms5Anita Shah6Pål André Holme7Lone H. Poulsen8Karina Meijer9Mindy Simpson10Maria Elisa Mancuso11Center for Bleeding and Clotting Disorders, University of Minnesota Medical CenterNational Hemophilia Center, Sheba Medical CenterNational Hemophilia Center, Sheba Medical CenterRegional Reference Center for Coagulation Disorders, Federico II University HospitalHemophilia Treatment Center, UC Davis Medical CenterBayer AGBayerDepartment of Haematology, Oslo University Hospital and Institute of Clinical Medicine, University of OsloThe Haemophilia Centre, Aarhus University HospitalUniversity Medical Center GroningenRush Hemophilia and Thrombophilia Center, Rush University Medical CenterCenter for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research HospitalAbstract Damoctocog alfa pegol (BAY 94-9027, Jivi®), is a site-specifically PEGylated, extended half-life recombinant factor VIII (FVIII) that is approved in several European and non-European countries for on-demand treatment and prophylaxis of bleeding in previously treated patients aged ≥ 12 years with hemophilia A. Reliable measurements can be obtained using most one-stage and chromogenic FVIII assays over a wide concentration range. The efficacy, safety and pharmacokinetics (PK) of damoctocog alfa pegol have been studied extensively in the PROTECT VIII clinical trials, and its long-term safety and effectiveness profile is continuing to build through observational and interventional real-world studies. The PK of damoctocog alfa pegol was shown to be improved as compared with that of sucrose-formulated rFVIII (rFVIII-FS, Kogenate®), and was also demonstrated to be non-inferior to and, for some variables, more favorable than rFVIII-Fc fusion protein, efmoroctocog alfa (Elocta®; NCT03364998), rurioctocog alfa pegol (BAX 855, Adynovate®/Adynovi®; NCT04015492), and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, Advate®; NCT02483208). Damoctocog alfa pegol was generally well tolerated and none of the patients in any of the clinical trials, including the PROTECT VIII clinical program, HEM-POWR, or ongoing single-center studies, developed FVIII inhibitors. Efficacy for perioperative hemostasis has been demonstrated. Low bleeding rates were achieved across the studies, with twice weekly, every 5-day and every 7-day prophylaxis offering patients ≥ 12 years and their clinicians the chance to tailor treatment to individual needs and lifestyles, while maintaining long-term protection from bleeds and their consequences. Graphical Abstracthttps://doi.org/10.1007/s40268-024-00481-7 |
| spellingShingle | Mark T. Reding Shadan Lalezari Gili Kenet Giovanni Di Minno Jonathan Ducore Alexander Solms Anita Shah Pål André Holme Lone H. Poulsen Karina Meijer Mindy Simpson Maria Elisa Mancuso Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review Drugs in R&D |
| title | Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review |
| title_full | Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review |
| title_fullStr | Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review |
| title_full_unstemmed | Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review |
| title_short | Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review |
| title_sort | damoctocog alfa pegol a pegylated b domain deleted recombinant extended half life factor viii for the treatment of hemophilia a a product review |
| url | https://doi.org/10.1007/s40268-024-00481-7 |
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