News Feature | August 28, 2014

Extended-Release Form Of Baxter's Advate Succeeds In Clinical Study

By Suzanne Hodsden

Baxter announced that Phase III studies of BAX 855, a full-length, longer-acting recombinant factor VIII developed to increase the half-life of ADVATE, led to a 95 percent reduction in annualized bleeding rate in patients with hemophilia A.

The CDC estimates that over 20,000 people in the U.S. suffer from hemophilia A, a genetic disease resulting from a faulty factor VIII gene, a clotting protein. Symptoms of hemophilia A include spontaneous bleeding, GI hemorrhage, frequent nosebleeds, or prolonged bleeding from cuts, scrapes, or surgeries.

Hemophilia A can be treated one of two ways. The first way, called on-demand treatment, seeks to treat patients only when bleeding problems occur.  The second method — prophylactic therapy — requires regular infusions to prevent potentially life-threatening issues.

ADVATE has been on the market for the past 10 years and has shown success in both on-demand and prophylactic treatments.

While the prophylactic therapy is considered the safest course of treatment, it is not the most convenient. According to the National Hemophilia Foundation (NHF) guidelines, traditional hemophilia A prophylactic therapy involves infusions every other day, amounting to approximately 150 to 180 infusions per year.

BAX 855 uses Nektar Therapeutics proprietary technology to extend the life of ADVATE (1.4-1.5 times) in the body and allows for less frequent doses — only twice per week.

The clinical trial found that prophylactic treatment using BAX 855 saw a 95 percent reduction in annual occurrences of bleeding compared to patients being treated on-demand.  Additionally, the clinical trial found no serious adverse reactions to the new form of the drug.

This most recent clinical study was conducted only on patients over the age of twelve, but Baxter indicated their goal of initiating another clinical study to test the efficacy and safety of the drug in younger children.

Reuters reports that Baxter proceeded with clinical research despite the FDA approval of Biogen Idec’s ELOCTATE earlier this year. ELOCTATE is a similar long-lasting treatment for hemophilia A. Further studies will show how the two different treatments compare.

John Orloff, VP and global head of research and development at Baxter states, “We look forward to advancing the BAX 855 program to U.S. regulatory submission by the end of this year.”