FDA Approves Baxter's Obizur For Acquired Hemophilia A

By Cyndi Root

Patients with acquired hemophilia A (AHA), a rare bleeding disorder, have a new treatment, as the Food and Drug Administration (FDA) has approved Baxter’s Obizur. Baxter announced the approval in a press release, stating that the Antihemophilic Factor (Recombinant), Porcine Sequence allows physicians to manage the treatment by measuring factor VIII activity levels. The FDA’s approval was based on a clinical trial showing 100 percent clinical response in 29 patients.

Dr. Rebecca Kruse-Jarres, the clinical trial's principal investigator, said, "Importantly, this new option to treat bleeding episodes will enable us to measure factor VIII levels, thus giving us an objective marker of hemostasis that can guide dosing and prevent overdosing."

Obizur

Obizur is derived from porcine (pig) FVIII, which is similar to human FVIII but is less likely to be affected by antibodies. Patients with AHA are rare but their condition is often life-threatening due to an autoantibody directed against coagulation factor VIII. Half of cases occur spontaneously or are due to unknown pathogenesis, while other cases occur due to drug use, malignancies, autoimmune conditions, or pregnancy. Obizur treatment raises factor VIII levels to temporarily aid in blood clotting. Baxter intends to make Obizur commercially available in the U.S. soon and continue pursuing regulatory approval in Europe and Canada.

Baxter’s Hemophilia Treatments

Baxter has 60 years of experience in hemophilia treatments and a broad portfolio of hemophilia treatments, including the newly approved Advate [Antihemophilic Factor (Recombinant)], FDA-approved in April 2014. The Wall Street Journal reports that Baxter’s hemophilia products were 58 percent of its business, $3.4 billion in 2013. The company recently made moves to support its hemophilia efforts, including spinning off a new company, Baxalta, and opening a manufacturing facility in Singapore for hemophilia treatments.

To support the Baxalta spin-off, Baxter is forming a new R&D center near Boston, MA. Baxter scientists at the facility will use gene therapy and biosimilar technology to focus on immunology and hematology. Currently in Singapore, the biologics facility processes Advate for hemophilia A patients.

The European Medicines Agency (EMA) approved the facility for processing Advate for supply to the European Union, Iceland, and Norway. Rixubis for hemophilia B is also manufactured there. Upon regulatory approval, Baxter intends to manufacture BAX 855, a PEGylated form of Baxter's rFVIII product Advate, based on the Advate manufacturing process.