The US Food and Drug Administration (FDA) has accepted Bayer’s biologics licence application (BLA) filing for BAY94-9027 to treat haemophilia A in adults and adolescents 12 years of age and above.
Haemophilia A is the most common type of haemophilia, in which blood clotting is affected either due to a lack or defect of coagulation Factor VIII (FVIII), a protein required to form blood clots.
Bayer’s investigational agent BAY94-9027 is an extended half-life site-specifically PEGylated recombinant human Factor VIII compound that is used for the treatment of the largely inherited disease.
Bayer Medical Affairs Americas senior vice-president and head Dr Dario Mirski said: “The filing acceptance for BAY94-9027 by the FDA represents a milestone to Bayer in its commitment to seeking potential treatments to address the unmet needs of people living with haemophilia A.”
The BLA submission is based on the results obtained from the pivotal multicentre, multinational, partially randomised, open-label Phase II / III PROTECT VIII clinical trial.
With four treatment arms, the trial was conducted to study the safety and efficacy of BAY94-9027 in previously treated adults and adolescents with severe haemophilia A.
In three of the prophylactic treatment arms, efficacy and safety of the therapy was evaluated when used once every seven days, once every five days or twice per week.
The therapy is developed to potentially prolong the FVIII activity in blood while preserving coagulation activity using site-specific PEGylation technology, where a Polyethylene glycol (PEG) molecule is consistently attached to the factor VIII protein at a specific site.
An increased half-life recombinant factor VIII treatment may facilitate reduced frequency of infusions for patients affected with haemophilia A.