Journal Article

Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review

Shah, Anita, Di Minno, Giovanni, Reding, Mark T., Kenet, Gili, Simpson, Mindy, Ducore, Jonathan, Meijer, Karina, Poulsen, Lone H., Lalezari, Shadan, Solms, Alexander, Holme, Pål André, Mancuso, Maria Elisa

2024

Overview

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.

• Publisher: Springer Nature B.V,Springer International Publishing,Adis, Springer Healthcare
• Subject: Cell culture / Disease prevention / Factor VIII - administration & dosage / Factor VIII - adverse effects / Factor VIII - pharmacokinetics / Factor VIII - therapeutic use / Half-Life / Hemophilia / Hemophilia A - drug therapy / Humans / Laboratories / Manufacturing / Pharmacokinetics / Polyethylene glycol / Polyethylene Glycols - administration & dosage / Polyethylene Glycols - chemistry / Polyethylene Glycols - pharmacokinetics / Product reviews / Proteins / Randomized Controlled Trials as Topic / Reagents / Review