FDA approves Baxter's FEIBA for prophylactic treatment of hemophilia patients with inhibitors

Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) granted approval of Baxter's FEIBA [Anti-Inhibitor Coagulant Complex], the first and only FDA-approved treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B who have developed inhibitors.

Inhibitor development is considered one of the most serious complications associated with hemophilia treatment today. As many as one-third of previously untreated patients with severe or moderately severe hemophilia A are at risk for developing inhibitors, which are antibodies produced by the body's immune system in response to factor replacement treatment. The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications.

The approval is based on data from a pivotal Phase III study, known as FEIBA PROOF, in which treatment with a FEIBA prophylactic regimen showed a 72 percent reduction in median annual bleed rate (ABR) compared to treatment with an on-demand regimen. In the intent-to-treat (ITT) analysis, three of the 17 (18%) adult patients in the prophylactic arm reported no bleeding episodes. The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

''The PROOF study demonstrated that a prophylactic regimen with FEIBA can significantly reduce the rate of bleeding episodes, as compared to an on-demand regimen, in hemophilia patients with inhibitors. This is important among these patients who often have difficult-to-treat bleeds and are at risk of additional complications,'' said Steven Pipe, M.D., Director, Division of Pediatric Hematology and Oncology, at the C.S. Mott Children's Hospital at the University of Michigan. ''This FDA approval of a prophylactic regimen should change the way physicians think about managing hemophilia with inhibitors, validating FEIBA prophylaxis as an effective new option to treat their patients.''

Pro-FEIBA, a prospective investigator-initiated, randomized, crossover study published in the New England Journal of Medicine in 2011 showed a 62 percent reduction in all bleeding episodes with FEIBA prophylaxis compared to an on-demand regimen. Together with the data from the FEIBA PROOF study, the results provide valuable evidence that prophylaxis with FEIBA can significantly reduce bleeding rates when compared to on-demand treatment.

''FEIBA has been an effective treatment for hemophilia patients with inhibitors for more than 35 years as an on-demand treatment. FEIBA was first licensed in the US as FEIBA VH and then as FEIBA NF. This additional indication for prophylactic treatment is aimed at reducing the number of bleeds among this patient population,'' said Ludwig Hantson, Ph.D., president of Baxter's BioScience business. ''This latest approval reflects our legacy of offering new approaches to help reduce the disease burden of hemophilia, and supports Baxter's ongoing commitment to pursuing a bleed-free world.''

FEIBA is approved in more than 60 countries worldwide and is indicated for prophylaxis in more than 40 countries.

FEIBA PROOF Study Details

The prospective, open label, randomized, multi-center, two-arm parallel study investigated the efficacy and safety of FEIBA prophylactic treatment compared to on-demand treatment in 36 patients with hemophilia A or B with inhibitors over a 12-month period. The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. The occurrence of hepatitis B surface antibodies has been seen in other plasma-derived products and could be due to the passive transfer of antibodies following FEIBA treatment. None of the subjects showed any signs of active hepatitis B infection.

Indications and Detailed Important Risk Information for FEIBA [Anti‐Inhibitor Coagulant Complex]

FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:

• Control and prevention of bleeding episodes
• Perioperative management
• Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation Factor VIII or coagulation Factor IX.