Bayer has submitted an application for marketing authorization for its extended half-life site-specifically PEGylated recombinant human Factor VIII (BAY94-9027, damoctocog alfa pegol) for the treatment of Hemophilia A in Japan. The regulatory submission is essentially based on the results from the PROTECT VIII trial. In that trial, BAY94-9027 showed protection from bleeds with dosing intervals when used prophylactically once every seven days, once every five days, and twice per week. Based on these data Bayer submitted BAY94-9027 for marketing authorization in this indication in the United States and Europe earlier this year already.
“Hemophilia A, affects approximately 5,000 patients in Japan. These patients are nowadays living longer and more fully lives thanks to Factor VIII replacement therapies,” said Dr. Joerg Moeller, member of the Executive Committee of Bayer AG's Pharmaceutical Division and Head of Development. “Besides minimizing the impact of this life-long disease, newer treatment options can reduce treatment burden and allow patients to live more normal, active lifestyles – thereby making a significant difference in their lives.”
In the PROTECT VIII study, BAY94-9027 provided good protection from bleeds when used prophylactically once every seven days, once every five days, and twice per week. BAY94-9027 was also effective for control of bleeding during surgical procedures, and treatment of all bleeds, with the vast majority of bleeding events being resolved with one or two infusions.
The standard-of-care for hemophilia A is factor VIII replacement therapy, which needs to be regularly infused into the patient’s vein to maintain factor levels high enough to prevent bleeding into joints, muscles or other organs. Due to the short half-life of most currently marketed factor VIII products, prophylaxis may require treatment as often as every other day or three times per week. BAY94-9027 is engineered to prolong activity in the body while preserving full coagulation activity through PEGylation, where a PEG (Polyethylenglycol) molecule is attached to the factor VIII protein at a specific site. PEGylation technology is utilized to prolong the time of drug circulation in the blood.