BAY94 9027,a long-acting site-specifically PEGylated recombinant human Factor VIII is filed at FDA for treatment of Hemophilia A.-Bayer

Bayer AG announced the submission of a Biologics License Application (BLA) with the FDA for its long-acting site-specifically PEGylated recombinant human Factor VIII (BAY94 9027) for the treatment of Hemophilia A. The regulatory submission is essentially based on the results from the PROTECT VIII trial.

In the PROTECT VIII 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. 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 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 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 consistently attached to the factor VIII protein at a specific site. PEGylation technology is utilized to prolong the time of drug circulation in the blood.