FDA Approves Baxter’s Treatment for Rare Hemophilia

Baxter International said it will launch its hemophilia drug Obizur in the next few months, after the FDA approved the product late last week.

Obizur (recombinant porcine antihemophilic factor) is indicated to treat bleeding in adults with acquired hemophilia A, a rare form of hemophilia that is autoimmune rather than genetic. Patients with the disease produce antibodies to their own clotting factor VIII.

While most replacement clotting factors given to hemophiliacs are human, Baxter’s comes from a pig. Pig factor is similar to human factor, but less likely to provoke an antibody attack, Baxter says.

According to the Illinois-based company, Obizur is the first porcine recombinant clotting factor that allows doctors to manage safety and efficacy not only through traditional clinical assessments, but also by monitoring factor VIII activity as a biomarker to measure bleeding levels. In a global clinical trial, all 28 patients showed improvement, with bleeding stopped or reduced within 24 hours of infusion.

Obizur received the FDA’s orphan drug designation, which is given to drugs that treat conditions affecting less than 200,000 patients in the U.S. It currently is awaiting approval in the European Union and Canada, according to the company. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.