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ARUP Laboratories Gets FDA Approval for Hemophilia Gene Therapy CDx

NEW YORK – ARUP Laboratories said Thursday it gained US Food and Drug Administration approval for its companion diagnostic to help identify which patients are eligible for treatment with a gene therapy for adults with severe hemophilia A.

The Salt Lake City-based firm's AAV5 DetectCDx is an electrochemiluminescence antibody assay for detection of antibodies in human plasma that bind to adeno-associated virus serotype 5 (AAV5). It was developed in collaboration with BioMarin Pharmaceutical, which received contemporaneous approval for its Roctavian gene therapy for adults who have hemophilia A (congenital factor VIII) deficiency with FVIII activity of less than 1 IU/dL and without antibodies to adeno-associated virus serotype 5.

Because BioMarin's gene therapy uses AAV5 to deliver a functional copy of the FVIII gene, the AAV5 DetectCDx test determines whether a patient has anti-AAV5 antibodies that could reduce treatment efficacy. ARUP noted that it is the only laboratory providing the test in the US.

"It is extremely satisfying to see the effort and dedication of both teams come to fruition after a successful and long-standing collaboration," ARUP Senior Director of Companion Diagnostics Emily Coonrod said in a statement.