FDA Files ARUP Laboratories' AAV5 CDx for Hemophilia A Gene Therapy Treatment

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Feb 20, 2020, 16:30 ET

SALT LAKE CITY, Feb. 20, 2020 /PRNewswire/ -- ARUP Laboratories announced today that the Food and Drug Administration (FDA) has filed ARUP's premarket approval application (PMA) for an AAV5 total antibody assay. The assay is intended as a companion diagnostic test for valoctocogene roxaparvovec, BioMarin's investigational gene therapy treatment for severe hemophilia A.

BioMarin partnered with ARUP to develop this test. ARUP is a laboratory with 35 years of experience supporting hospitals, physicians, and patients with unparalleled quality and service including two prior FDA companion diagnostic approvals. ARUP's experience in developing FDA-approved, single-source companion diagnostic tests is unique among reference laboratories.

ARUP's AAV5 total antibody assay is a simple blood test that is intended to help identify patients most likely to respond to AAV5-based gene therapy. BioMarin implemented the test in multiple clinical studies evaluating valoctocogene roxaparvovec treatment of hemophilia A patients without antibodies to AAV5. If approved, ARUP's AAV5 total antibody assay may be the first companion diagnostic test for any gene therapy product.

"We are delighted to have partnered with BioMarin on its hemophilia A gene therapy program," said ARUP CEO Sherrie Perkins, MD, PhD. "Working with BioMarin on the simultaneous submission of the diagnostic and gene therapy applications aligns with our corporate commitment to providing outstanding patient care through cutting-edge diagnostic testing."

ARUP Media Contacts:
Cyndee Holden, 801-583-2787, ext. 3318, [email protected]
Peta Owens-Liston, 801-583-2787, ext. 3635, [email protected]

About ARUP Laboratories
Founded in 1984, ARUP Laboratories is a leading national reference laboratory and a nonprofit enterprise of the University of Utah and its Department of Pathology. ARUP offers more than 3,000 tests and test combinations, ranging from routine screening tests to esoteric molecular and genetic assays. ARUP serves clients across the United States, including many of the nation's top university teaching hospitals and children's hospitals, as well as multihospital groups, major commercial laboratories, group purchasing organizations, military and other government facilities, and major clinics. In addition, ARUP is a worldwide leader in innovative laboratory research and development, led by the efforts of the ARUP Institute for Clinical and Experimental Pathology^®.

About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

SOURCE ARUP Laboratories