Obizur is a coagulation factor VIII of pig origin approved for the treatment of bleeding episodes in adults with acquired hemophilia. Originally developed by Baxalta, it is now marketed by Takeda.

The U.S. Food and Drug Administration (FDA) approved Obizur in 2014. A year later, the European Commission (EC) granted the authorization for its use in the E.U.

How Obizur works

Patients with acquired hemophilia have blood clotting problems due to the presence of antibodies that target the clotting factor VIII. Factor VIII is one of the proteins required for normal blood clotting. Patients with acquired hemophilia experience bleeding in the internal organs, muscles, and joints.

Obizur is a recombinant factor VIII that is synthesized by a gene that carries the instructions to make factor VIII in pigs. Obizur mimics the functioning human factor VIII but it slightly different structurally. Therefore, it can replace the human factor VIII without being recognized and deactivated by the antibodies in the patients’ blood and help in blood clotting.

Obizur in clinical trials

The approval of Obizur for the treatment of acquired hemophilia was based on data from a Phase 2/3 multicenter clinical trial (NCT01178294). This international study assessed the safety and efficacy of Obizur in 28 patients with acquired hemophilia (median age 70 years). The patients were initially treated with 200 U/kg of Obizur followed by additional doses depending on their response and factor VIII activity levels in the blood. Patients also received immunosuppressive treatment during the study.

Within 24 hours of treatment, Obizur either stopped or reduced bleeding in all patients, meeting the primary endpoint of a positive clinical response. Compared to the start of the study, a significant increase in factor VIII activity levels was reported in all the patients after 24 hours of treatment. No adverse effects were reported.

A post-marketing multi-center study (NCT02610127) is surveilling at least 60 adult patients with acquired hemophilia who are being treated with Obizur to assess the effectiveness and safety of the treatment in a real-world setting. The ongoing observational study is an attempt to collect patient data post-FDA approval to continue monitoring any safety concerns associated with Obizur use.

An ongoing non-interventional study (NCT03199794) is evaluating the effectiveness and safety of Obizur prescribed to people with acquired hemophilia by their treating physicians. The duration of this international study is 180 days. This is a real-life study enrolling approximately 50 acquired hemophilia patients, age 18 and older, in Austria, Germany, Italy, and the Netherlands.

Other information

Obizur may cause a number of side effects. These include:

  • Headache
  • Nausea or vomiting
  • Chills
  • Burning at the site of injection
  • Angioedema
  • Hives
  • Low blood pressure
  • Chest tightness, tingling, or rapid heartbeat
  • Breathing problems like wheezing

Some patients may develop inhibitory antibodies to the porcine factor VIII, making the treatment ineffective. In such cases, it is recommended to consider alternate therapeutic options in consultation with the patient’s care team.

Obizur is not recommended for patients with congenital hemophilia A or von Willebrand disease.

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Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified healthcare providers with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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Vijaya Iyer is a freelance science writer for BioNews Services. She has contributed content to their several disease-specific websites, including cystic fibrosis, multiple sclerosis, muscular dystrophy, among others. She holds a PhD in Microbiology from Kansas State University, where her research focused on molecular biology, bacterial interactions, metabolism, and animal models to study bacterial infections. Following the completion of her PhD, Dr. Iyer went on to complete three postdoctoral fellowships at Kansas State University, University of Miami and Temple University. She joined BioNews Services to utilize her scientific background and writing skills to help patients and caregivers remain abreast with important scientific breakthroughs.
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