Bayer said today it has submitted to the FDA a Biologics License Application (BLA) for BAY94-9027 (damoctacog alfa pegol), a long-acting site-specifically PEGylated recombinant human Factor VIII designed to treat hemophilia A up to once every seven days.
“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,” Joerg Moeller, M.D., member of the executive committee of Bayer AG's Pharmaceutical Division and head of development, said in a statement.
The BLA is supported by positive data from the Phase III PROTECT VIII (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII) trial. Bayer trumpeted positive results from PROTECT VIII in 2014, saying the study showed BAY94-9027 met its primary endpoint of protection from bleeding with fewer infusions.
According to Bayer, BAY94-9027 protected patients from bleeds when used prophylactically once every seven days, once every five days, and twice per week. The company added that the treatment was also effective for treatment of acute and breakthrough bleeds, with nearly all (91%) of events were resolved with one or two infusions.
BAY94-9027 is engineered to prolong activity in the body while preserving full coagulation activity through PEGylation. A polyethylenglycol (PEG) molecule is consistently attached to the Factor VIII protein at a specific site, with the aim of prolonging the time of drug circulation in the blood.
Bayer’s BLA for BAY94-9027 comes more than a year after the company won approvals in the U.S. and Europe for Kovaltry® Antihemophilic Factor (Recombinant). Kovaltry, an unmodified, full-length Factor VIII compound also known as octocog alfa or BAY 81-8973, is a next-generation version of Bayer’s flagship hemophilia A treatment Kogenate® FS Antihemophilic Factor (Recombinant) that is indicated for the treatment of hemophilia A in children and adults.
Last year, Bayer generated €1.166 billion ($1.38 billion) in sales from Kogenate and Kovaltry, up 1% from 2015. The company did not break out sales of the drugs in its 2016 Annual Report.
“Since introducing Kogenate around 25 years ago, Bayer has been committed to continuously improving disease management for people living with hemophilia A,” Dr. Moeller added.
According to Bayer, hemophilia affects approximately 400,000 people worldwide, with an estimated frequency of 1 in 5000 male live births, affecting approximately 30,000 in Europe, and 14,000 in the U.S. today.
BAY94-9027 is the second Bayer-developed hemophilia A drug to make news in recent days. On Friday, Regenxbio agreed to acquire Dimension Therapeutics for approximately $85 million—a deal that includes Dimension’s DTX201, a gene therapy candidate for hemophilia A being developed and commercialized through an up-to-$252 million collaboration with Bayer launched in 2014.