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FDA Grants Approval to ARUP Laboratories' AAV5 DetectCDx™ as the First Immunoassay Companion Diagnostic for Gene Therapy

Friday, June 30, 2023

ARUP Laboratories announced today that the U.S. Food and Drug Administration (FDA) has granted approval for AAV5 DetectCDx™ as a companion diagnostic test. The test is intended to assist in the selection of adult patients eligible for treatment with ROCTAVIAN™, a gene therapy developed by BioMarin Pharmaceutical Inc. ROCTAVIAN™ received FDA approval for the treatment of adults with severe hemophilia A, a genetic bleeding disorder caused by a deficiency in coagulation factor VIII (FVIII).

AAV5 DetectCDx™ is the first companion diagnostic immunoassay specifically designed for a gene therapy product to receive FDA approval. ARUP Laboratories collaborated with BioMarin in the development of this diagnostic test. As part of the approval, ARUP Laboratories will be the sole provider of the test in the United States.

Hemophilia A patients may experience life-threatening bleeding due to a lack of clotting protein. ROCTAVIAN™ aims to deliver a functional copy of the FVIII gene through an adeno-associated virus serotype 5 (AAV5) vector, potentially reducing the need for ongoing treatment. AAV5 DetectCDx™ will be used to identify patients without antibodies to AAV5, which could hinder the effectiveness of the gene therapy. Only patients who test negative for anti-AAV5 antibodies using AAV5 DetectCDx™ will be eligible for treatment with ROCTAVIAN™ according to the current FDA approval.

The FDA approval of AAV5 DetectCDx™ is the result of a collaborative effort between ARUP Laboratories and BioMarin. The diagnostic test was evaluated in various clinical studies, demonstrating its potential

benefits in treating severe hemophilia A in adults. This marks the third FDA-approved companion diagnostic test developed by ARUP Laboratories. Previously approved tests from ARUP Laboratories aid in the selection of patients with rare hematologic disorders for specific treatments under the Humanitarian Device Exemption (HDE) program.

AAV5 DetectCDx™, also known as the AAV5 Total Antibody Assay for ROCTAVIAN (valoctocogene roxaparvovec-rvox) Eligibility in Hemophilia A, is a qualitative in vitro diagnostic test that utilizes electrochemiluminescence. It is designed to detect antibodies in human plasma collected in 3.2% sodium citrate, specifically targeting adeno-associated virus serotype 5 (AAV5). The test is used to determine whether adult hemophilia A patients are eligible for treatment with ROCTAVIAN™. Patients who test positive for anti-AAV5 antibodies (result of "Detected") are not eligible for treatment, while those who test negative (result of "Not Detected") qualify for ROCTAVIAN™. The AAV5 DetectCDx™ assay is performed exclusively at ARUP Laboratories.



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