FDA Approves Hemophilia Drug FEIBA for Bleeding Prophylaxis

Miriam E. Tucker

Disclosures

December 23, 2013

The US Food and Drug Administration (FDA) has approved a new indication for Baxter's anti-inhibitor coagulant complex FEIBA as the first-ever routine prophylaxis to prevent bleeding in patients with hemophilia A or B who have developed inhibitors.

About a third of previously untreated patients with severe or moderately severe hemophilia A develop inhibitors, or antibodies produced in response to clotting factor replacement. The presence of inhibitors complicates treatment and increases the risk for complications such as joint damage.

FEIBA is already indicated for the control of spontaneous bleeding episodes and to cover surgical interventions in patients with hemophilia A and hemophilia B who have inhibitors. Now it will be available for routine use as prophylaxis.

Data supporting FEIBA's prophylaxis indication came from a pivotal phase 3 trial called FEIBA PROOF, which enrolled 36 patients with hemophilia A or B and inhibitors. At 12 months, FEIBA prophylaxis produced a 72% reduction in annual bleed rate compared with on-demand treatment (7.9% vs 28.7%). In the intent-to-treat analysis, 3 of 17 patients who received FEIBA had no bleeding episodes.

The most common adverse reactions, seen in more than 5% of the study participants, were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positivity, nausea, and vomiting. Serious adverse reactions seen with FEIBA in all trials include hypersensitivity reactions and thromboembolic events (stroke, pulmonary embolism, and deep vein thrombosis).

Before FEIBA PROOF, a study called Pro-FEIBA also showed prophylactic efficacy for the drug. In this 6-month prospective randomized investigator-initiated randomized crossover study in which 26 of 34 patients completed treatment, there was a significant 62% reduction in all bleeding episodes with FEIBA compared with an on-demand regimen (P < .001).

Pro-FEIBA, which was funded by Baxter Bioscience and published in the New England Journal of Medicine in 2011, also showed a 61% reduction in hemarthroses (P < .001) and a 72% reduction in target-joint bleeding (P < .001). Of 33 patients who received at least 1 FEIBA infusion, 1 had an allergic reaction to it ( N Engl J Med. 2011;365:1684-1692).

''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,'' Steven Pipe, MD, director of the Division of Pediatric Hematology and Oncology at the C.S. Mott Children's Hospital at the University of Michigan, Ann Arbor, said in a Baxter statement.

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