Bayer submits BLA in the for BAY94-9027 – a long-acting factor VIII for the treatment of hemophilia A
Pivotal studies with BAY94-9027 showed that bleed protection was achieved with extended dosing intervals.
Bayer has submitted a Biologics License Application (BLA) with the UFDA 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 that trial, BAY94-9027 showed protection from bleeds with dosing intervals when used prophylactically once every 7 days, once every 5 days, and twice per week.
“Since introducing Kogenate around 25 years ago, Bayer has been committed to continuously improving disease management for people living with Hemophilia A,” said Dr Joerg Moeller, member of the Executive Committee of Bayer AG's Pharmaceutical Division and Head of Development. “The filing of BAY94-9027 brings us one step closer to providing a therapeutic option with additional benefits for patients who decided to have a more active lifestyle.”
In the PROTECT VIII study, BAY94-9027 provided good protection from bleeds when used prophylactically once every 7 days, once every 5 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.
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