Baxter International ($BAX) reports that its pivotal Phase III study comparing routine preventive use versus on-demand treatment using its anti-inhibitor coagulant complex FEIBA NF for hemophilia passed with flying colors, setting up plans to file for an approval soon.
Routine prophylaxis of the therapy led to a 72.5% reduction in the annual bleed rate among hemophiliacs in the late-stage trial, Baxter noted, adding that it is prepping a biologics license application to be filed in the first quarter of this year.
"Treatment with FEIBA NF resulted in a significant reduction in annual bleed rate of all types of bleeds in the prophylaxis arm as compared to the on-demand arm," said lead investigator Dr. Sandra Antunes. "Three of the 17 intent to treat patients (17.6 %) in the prophylaxis arm did not experience any bleeding episodes during the study, and this is very significant for hemophilia patients with inhibitors."
Baxter says that up to one in three hemophiliacs develop an inhibitor to a product used to treat or prevent bleeding episodes. The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications such as joint damage.
The most commonly reported adverse reactions in the study were hypersensitivity, dizziness, headache, rash, hypotension and hepatitis B surface antibody positive laboratory test result, investigators noted. A temporary spike in hepatitis B surface antibodies was seen, but none of the patients exhibited any sign or symptom of hepatitis B infection.
"One of the greatest remaining challenges in the management of hemophilia is the development of inhibitors, which can lead to more difficult-to-control and sometimes life-threatening bleeding. The FEIBA NF prophylaxis study demonstrates Baxter's dedication to providing treatment options to the hemophilia community, including effective inhibitor management," said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter's BioScience business.
- here's the press release