New Kit Demonstrates Clinical Validity as Measure of FVIII Inhibitors in Hemophilia A

Precision BioLogic revealed promising data on a new kit for the measurement of FVIII inhibitors (factor VIII) in patients with hemophilia A.

The results were presented at the International Society on Thrombosis and Haemostasis’ Scientific and Standardization Committee (SSC) meeting July 18-21 in Dublin, Ireland.

Researchers presented their findings in a poster titled, “Emicizumab Impact on Factor VIII Inhibitor Determination in Plasma Samples from Persons with Hemophilia A (PwHA) Using a New Kit for Modified Nijmegen-Bethesda Assay (MNBA).”

Hemophilia A is a bleeding disorder caused by low levels of FVIII in the blood. The standard treatment for patients is FVIII replacement therapy through intravenous administration of recombinant (lab made) FVIII (rFVIII) or plasma-derived FVIII (pdFVIII) concentrates.

Unfortunately, up to 30% of hemophilia A patients develop inhibitors to the clotting factor concentrates. While routine blood tests allow for the identification of anti-factor FVIII antibodies, specialized testing is needed to confirm and quantify levels to effectively adjust treatment.

Currently, there is no gold standard for the testing of FVIII inhibitors, so there is a need for a standardized and improved assay that tests for levels of FVIII inhibitors.

Precision BioLogic collaborated with Roche and Genentech, a member of the Roche Group, to develop a new Modified Nijmegen-Bethesda Assay (MNBA) and determined its validity in clinical use.

The latest results from the study indicate that this MNBA test is suitable for measuring FVIII inhibitor levels using plasma (blood) samples containing Roche’s Hemlibra — an antibody that recognizes factor IXa and factor X and brings them together to activate the body’s natural clotting pathway, thereby surpassing the need for factor VIII.

Hemlibra has been approved by both the U.S. Food and Drug Administration and the European Commission for preventive treatment of hemophilia A patients with factor VIII inhibitors.

The data disclosed at the SSC meeting in Dublin builds on previous findings that were released at the Thrombosis & Hemostasis Societies of North America (THSNA) 2018 Summit in San Diego, California, and the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, Scotland.

“We’re seeing promising advancements in the treatment of bleeding disorders,” Paul Empey, president and CEO of Precision BioLogic, said in a press release. “We are hopeful that combining accurate diagnosis and monitoring with potential advancements could improve the quality of life for people with bleeding disorders. Precision BioLogic is proud to be at the forefront of this promising research.”

Precision BioLogic’s newly formulated MNBA kit was developed using four main components: imidazole-buffered pooled normal plasma, imidazole-buffered bovine serum albumin, positive FVIII inhibitors control, and FVIII inhibitor-free human plasma.

Precision BioLogic plans to commercialize the MNBA kit and will apply for clearance from global regulatory authorities starting in late 2018.