The US Food and Drug Administration (FDA) has approved Baxter’s FEIBA [anti-inhibitor coagulant complex] for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A or B patients with inhibitors.
At present, inhibitor development is considered one of the most serious complications with regard to hemophilia treatment.
Around 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.
According to the company, patients with inhibitors have an increased risk of developing complications.
FDA approval is based on data from a Phase III trial called FEIBA PROOF, in which treatment with a FEIBA prophylactic regimen showed a 72% 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 adult patients in the prophylactic arm reported no bleeding episodes and the most frequently reported adverse reactions seen in >5% of subjects in the trial were anemia, diarrohea, hemarthrosis, hepatitis B surface antibody positive, nausea and vomiting.
In the trial, the serious adverse reactions observed with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.
CS Mott Children’s Hospital Division of Pediatric Hematology and Oncology director Steven Pipe said the PROOF study showed that a prophylactic regimen with FEIBA can significantly reduce the rate of bleeding episodes, as compared with 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," Pipe said.
"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."
The company said that Pro-FEIBA, a prospective investigator-initiated, randomised, crossover study published in the New England Journal of Medicine in 2011 showed a 62% reduction in all bleeding episodes with FEIBA prophylaxis compared to an on-demand regimen.
Along with the data from the FEIBA PROOF study, the results provide valuable evidence that prophylaxis with FEIBA can reduce bleeding rates compared with on-demand treatment, the company said.