US regulators are reviewing Bayer’s BAY94-9027, a longer-acting Factor VIII therapy designed to reduce the number of infusions necessary to prevent bleeds in patients with Haemophilia A.
The submission is based on data from the PROTECT VIII pivotal Phase II/III trial, which assessed the efficacy and safety of the treatment when used on-demand and prophylactically once every seven days, once every five days, or twice per week.
The study met its primary endpoint of protection from bleeds with fewer infusions, and was also shown to be effective for treating acute and breakthrough bleeds, with 91 percent of events resolving with one or two infusions.
BAY94-9027 is engineered to potentially prolong FVIII activity in the blood while preserving coagulation activity using site-specific PEGylation technology, where a PEG (Polyethylene glycol) molecule is consistently attached to the factor VIII protein at a specific site.
An extended half-life recombinant factor VIII therapy may result in reduced frequency of infusions for people living with hemophilia A, the firm noted.
The therapy has not yet been approved anywhere in the world but remains under review in Europe for adults and adolescents aged 12 years and over.
"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 hemophilia A," said Dario Mirski, MD, Senior Vice President and Head of Medical Affairs Americas, Bayer. "