FDA Receives Submission for Hemophilia A Drug BAX 855

An investigational, extended half-life recombinant factor VIII treatment for hemophilia A was submitted to the US Food and Drug Administration (FDA) for a biologics license application, Nektar Therapeutics and Baxter International Inc announced.

Submission for the approval of the drug, BAX 855, was based on positive results of a phase 3 study. In the global study, 137 previously treated patients aged 12 years or older either experienced a twice weekly prophylaxis or an on demand treatment. In the twice weekly prophylaxis group, patients experienced a 95 percent reduction in median annualized bleed rate compared to the on demand treatment group, 1.9 vs. 41.5, respectively. The drug also successfully treated bleeding episodes; of those, 96 percent were controlled with 1 or 2 infusions. The researchers reported no inhibitor development in patients after treatment with the drug as well as no serious adverse events such as hypersensitivity. The most common adverse reaction was headache, found in 3 patients.

More data is expected for presentation in the coming months, as the trial is ongoing. The researchers hope to soon branch out to test the drug on severe hemophilia A patients under the age of 12 years.

“The evidence from our late-stage controlled trial showcases that BAX 855 could become an important new treatment option for people with hemophilia A, expanding on more than a decade of experience with Advate,” John Orloff, MD, vice president and global head of research and development for Baxter BioScience, said in a press release. “BAX 855 represents our continued commitment to developing valuable new options for the hemophilia community that address the needs of patients around the world.”

Advate is a currently used rFVIII antihemophilic drug indicated for use in children and adults with hemophilia A. It is designed to control and prevent bleeding episodes, for perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. Advate is currently approved in 64 countries, including the US, and is advertised as the world’s most prescribed FVIII treatment.