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ARUP Laboratories Gains IVDR-CE Mark for Hemophilia Gene Therapy CDx

NEW YORK – ARUP Laboratories said Friday that its companion diagnostic to help identify which patients are eligible for treatment with a gene therapy for hemophilia A received CE marking under the European Union's In Vitro Diagnostic Regulation (IVDR).

The AAV5 DetectCDx electrochemiluminescence antibody assay is used to help select patients for treatment with BioMarin Pharmaceutical's Roctavian (roxaparvovec-rvox) gene therapy for severe hemophilia A. The test was developed in collaboration with BioMarin and has been CE marked since 2020 under the former In Vitro Diagnostic Medical Devices Directive. ARUP said that securing certification under IVDR will help ensure continued access to the test in the EU when all medical devices will need to meet IVDR requirements in May 2026.

The firm also secured in June US Food and Drug Administration approval for the companion diagnostic.

BioMarin's therapy uses an adeno-associated virus serotype 5 as a vector to deliver into patients a functional copy of the FVIII gene, and ARUP's companion diagnostic is used to detect in plasma anti-AAV5 antibodies that can inhibit treatment. The treatment can reduce or alleviate the need for chronic prophylactic treatment.

ARUP said it collaborated with BioMarin on achieving IVDR certification, and ARUP performs the test at its College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified reference laboratory in Salt Lake City.

"We are gratified to see the results of this colossal effort from both teams and to fulfill an unmet need for patients with rare diseases such as hemophilia A," ARUP PharmaDx Companion Diagnostics Group Manager Jennifer Granger said in a statement.