Baxter International Inc has submitted a new drug application (NDA) to Japan's Ministry of Health, Labour and Welfare for the approval of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment based on Antihemophilic Factor (Recombinant) (Advate) for patients over 12 years of age with haemophilia A.

"We continue to advance BAX 855 as a potential new treatment for haemophilia A patients around the world who are seeking options that support their individual needs," said John Orloff, managing director, vice president and global head of research and development for Baxter BioScience.

"With more than a decade of experience with Advate and an extensive global presence, our BAX 855 program represents our continued commitment to supporting the haemophilia community, particularly this week as we celebrate World haemophilia Day with our colleagues around the globe," said Orloff.

The submission follows the filing to the United States Food and Drug Administration (FDA) in late 2014 and is based on positive results from a prospective, global, phase 3 study of 137 previously treated patients.

The results, presented during the European Association for Haemophilia and Allied Disorders (EAHAD) meeting in February 2015, demonstrated that BAX 855 met its primary endpoint in the control and prevention of bleeding episodes and routine prophylaxis for patients who were 12 years or older.

Patients in a twice-weekly prophylaxis arm experienced a 95 per cent reduction in median annual bleed rate (ABR) as compared to those in the on-demand arm (1.9 vs. 41.5, respectively).

BAX 855 was also effective in treating bleeding episodes, 96 percent of which were controlled with one or two infusions. No patients developed inhibitors to BAX 855 and no treatment-related serious adverse events, including hypersensitivity, were reported. The most common product-related adverse reaction was headache (3 patients).

Baxter continues to advance a continuation study for patients who completed the pivotal trial and a phase 3 study among previously treated patients under the age of 12 with severe haemophilia A.

Once the pediatric study has been completed, the company expects to file for marketing authorization with the European Medicines Agency in 2016 and intends to seek post-approval label expansion in the US for previously-untreated pediatric patients.


BAX 855 is based on Advate, a full-length FVIII molecule with more than12 years of real-world experience.

Through a collaboration with Nektar Therapeutics, BAX 855 leverages proprietary pegylation technology designed to extend the duration of activity of the protein in the body.

This proprietary technology has been used for over 10 years in a number of approved medicines that treat chronic or serious conditions.

Advate is a recombinant antihemophilic factor indicated for use in children and adults with haemophilia A (congenital factor VIII deficiency or classic haemophilia) for control and prevention of bleeding episodes, perioperative management, routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Advate is not indicated for the treatment of Von Willebrand disease. Advate has a demonstrated efficacy and safety profile. Advate is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives.

Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated.

There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.

Advate is the world's most prescribed FVIII treatment. It is currently approved in 64 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with haemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions.